-
Subject Areas on Research
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1028. Use of Antisense Oligonucleotides To Restore Activity in Mutant p53
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104. Role of the HI Loop in the Adeno- Associated Virus (AAV) Life Cycle
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1064. Haute Couture Vectors: Solving Clinical Problems with Custom Designed AAV Vectors
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1088. Stem Cell Gene Therapy with No Pre-Conditioning for the ADA-Deficiency Patients Leads to Generalized Detoxification and Delayed, but Steady Hematological Reconstitution
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14. Next Generation AAV Vectors for Limiting Systemic Leakage and Improving Safety Following CNS Administration
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167. Genome Editing to Generate the First Mouse Model of Alpha-One Antitrypsin Deficiency, the Leading Cause of Genetic COPD
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2'-Deoxy-2'-fluoro-arabinonucleic acid: a valid alternative to DNA for biotechnological applications using charge transport.
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211. Development of Helper-Dependent Adenovirus (HDAd) Vectors for Gene Therapy in the Mouse Model for Glycogen Storage Disease Type Ia (GSD-Ia)
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219. Persistent Correction of Hyperphenylalaninemia Following Liver-Directed, rAAV2/8-Mediated Gene Therapy for Murine Phenylketonuria (PKU)
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258. Phase I/IIa Study of TT-034, a DNA-Directed RNA Interference (ddRNAi) Agent Delivered as a Single Administration for the Treatment of Subjects with Chronic Hepatitis C Virus (HCV)
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265. Polysialic Acid as a Novel Regulator of AAV Tropism in the Developing Brain
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30. Modulation of Intracellular Calcium Enhances AAV Transduction in the CNS
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30. Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID) Using a γ-Retroviral Vector
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302. Combinatorial Engineering of a Receptor Footprint on AAV Serotype 4 Yields Novel Vectors
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311. In Vivo Zinc Finger Nuclease-Mediated Targeted Integration of a Glucose-6-Phosphatase Transgene Enhances Biochemical Correction in Mice with Glycogen Storage Disease Type IA
-
339. Sustained, High-Level Expression of Human Acid Alpha-Glucosidase and Correction of Glycogen Storage Disease Type II (GSD II) with an Adeno-Associated Virus 8 (AAV2/8) Vector Containing a Liver-Specific Promoter
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38. Pharmacological Regulation of Vesicular Trafficking as a Strategy to Enhance Recombinant AAV Transduction
-
381. Prohypertrophic Agents Enhance the Response to Gene Therapy in Pompe Disease
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397. Correction of the Dystrophin Gene By the CRISPR/Cas9 System in a Mouse Model of Muscular Dystrophy
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41. U2 snRNP Spliceosome Proteins Block Recombinant AAV Vector Transduction
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42. Differential Transduction Profiles of AAV Vectors in a Mouse Model of Human Glycosylation
-
425. Translation of Enhanced Dendritic Cell Vaccines Using a CID-Inducible CD40 Receptor
-
427. Adenovirus Mediated GRP94/gp96 Expression in Treatment of Neuroblastoma
-
472. Low-Shear Sodding of an Implantable Expanded Polytetrafluoroethylene Scaffold with a Rat Adipose-Derived Stromal-Vascular Fraction
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482. Local and Systemic Gene Editing in a Mouse Model of Duchenne Muscular Dystrophy
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485. Targeted Epigenome Editing by CRISPR/Cas9-Based Repressors for Silencing of Distal Regulatory Elements
-
487. Liver-Targeted Gene Therapy in Glycogen Storage Disease Type Ia (GSD-Ia) Requires Widespread, Regulated Glucose-6-phosphatase
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49. Minimum Effective Dose of Liver-Targeted Gene Therapy for Pompe Disease Reduced 10-Fold from Prior Estimates
-
5 ' exon replacement by spliceosome mediated RNA trans-splicing
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504. Restoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular Dystrophy
-
563. Correction of Glycogen Storage Disease Type II by Systemic Delivery of an AAV2/8 Vector Containing a Muscle-Specific Promoter
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586. Anti-HIV-1 Neutralizing Antibody Gene Delivery to Rhesus Macaques and Rodents: A Novel Vaccination Approach
-
59. Multiplex Gene Activation by CRISPR/Cas9-Based Transcription Factors for the Direct Conversion of Fibroblasts to a Neuronal Phenotype
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61. Vascular Smooth Muscle Cell RNA Aptamers for the Treatment of Cardiovascular Disease
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66. Identification and Characterization of a B Cell Aptamer That Targets Diffuse Large B Cell Lymphoma (DLBCL) and Chronic Myelogenous Leukemia (CML)
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7. Adeno-Associated Virus Type 2 (AAV2) Contains an Integrin alpha5beta1 Recognition Sequence Essential for Viral Cell Entry
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701. Long-Term Complications of Glycogen Storage Disease Type IA in the Dog Model Treated With Gene Replacement Therapy
-
727. Characterizing the Adeno-Associated Virus 1 Sialic Receptor Binding Site and Its Overlap with Antigenic Epitopes
-
739. Bioluminescent Virion Shells: A Novel Tool for Visualization of Viral Vectors in Animal Tissue
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8. Further Characterization of U2 snRNP Mediated Restriction of AAV Vector Transduction
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83. Cell_Type Specific Delivery of siRNAs with Aptamer_siRNA Chimeras
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93. Development of AAVidua Vector for the Treatment of Cornea Associated Vision Loss in Bone Marrow Treated-Hurler Patients
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954. High Intensity Focused Ultrasound (HIFU) Induced Trans-Gene Activation in Solid Tumors
-
A Beautiful Mind and the Heart of an Explorer
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A CRISPR Screen Identifies Apical Polarity Determinant Crumbs 3 as an AAV Host Restriction Factor
-
A CRISPR/Cas9 Genome Wide Screen and Cryo-EM Structure Reveal Receptor Switching by a Synthetic AAV Variant
-
A CRISPRa Screen Identifies Transcription Factors That Can Silence or Activate rAAV Genomes
-
A Combination Immunosuppression Treatment with Bortezomib and an Anti-CD20 mAb Enables Successful Re-Administration of AAV8 Vector in Mice
-
A Human iPSC-Derived Neuron and Cardiomyocyte Platform for Assessing Novel Recombinant Adeno-Associated Viral (AAV) Vectors
-
A Newly Evolved AAV Variant Enables Potent Gene Transfer in Kidneys of Multiple Species
-
A Novel Approach for Rapid and Transient Depletion of Pre-Existing Neutralizing Antibodies Against AAV Vectors
-
A Novel Aptamer Targeting Agent for Prostate Cancer
-
A Novel Capsid for Smooth Muscle Pathology in Pompe Disease
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A Novel Human Liver Chimeric Mouse Model Lacking the Murine AAVR Gene for Validation of AAV Gene Therapy Vectors
-
A Single Intravenous Injection of an AAV-PHP. B Vector Encoding Human Acid alpha-Glucosidase Corrects Both Muscle and Brain Defects in Murine Pompe Disease
-
A Status Report on FDA Approval of Medical Devices Containing Nanostructured Materials.
-
A central role of RNA in developing therapeutic vaccines for cancer and infectious diseases
-
A clinical attempt to treat JAK3-deficient SCID using retroviral-mediated gene transfer to bone marrow CD34+ cells
-
A cone-plate apparatus for the in vitro biochemical and molecular analysis of the effect of shear stress on adherent cells
-
A cornucopia of strategies for the immunotherapy of cancer
-
A genetically engineered plasmid encoding a zinc finger VEGF-activating transcription factor induces angiogenesis in the rabbits with hind-limb ischemia
-
A kinetic analysis using fractals of cellular analyte-receptor binding and dissociation.
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A large U3 deletion causes increased in vivo expression from a nonintegrating lentiviral vector.
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A method for the organ explant culture of fish kidney tubules
-
A microfluidic system for investigation of extravascular transport and cellular uptake of drugs in tumors.
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A model for optimizing adenoviral delivery in human cancer gene therapy trials.
-
A need for effective adjuvants.
-
A new non-enzymatic method for isolating human intervertebral disc cells preserves the phenotype of nucleus pulposus cells.
-
A novel antidote-controlled anticoagulant reduces thrombin generation and inflammation and improves cardiac function in cardiopulmonary bypass surgery.
-
A novel conditionally replicating "armed" adenovirus selectively targeting gastrointestinal tumors with aberrant wnt signaling.
-
A novel thermostable branching enzyme from an extremely thermophilic bacterial species, Rhodothermus obamensis.
-
A numerical analysis of forces exerted by laminar flow on spreading cells in a parallel plate flow chamber assay.
-
A phase I trial of human gamma interferon transduced autologous tumor cells in patients with disseminated malignant melanoma.
-
A physical characterization of GAP A3 hybridoma cells: morphology, geometry, and mechanical properties.
-
A pressure-mediated nonviral method for efficient arterial gene and oligonucleotide transfer.
-
A quantitative method for measuring gene expression magnitude and volume delivered by gene therapy vectors.
-
A roadmap for interpreting (13)C metabolite labeling patterns from cells.
-
A small regulatory element from chromosome 19 enhances liver-specific gene expression.
-
A synthetic biology challenge: making cells compute.
-
A tumour specific targeted radiotherapy/gene therapy for the treatment of malignant disease
-
A year of successful cancer vaccines points to a path forward.
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AAV Capsid Dynamics at the 5-Fold Pore Controls Genome Release
-
AAV Gene Therapy Effectively Transduces the Central Nervous System and Prevents Polyglucosan Body Formation in Adult Polyglucosan Body Disease Mice
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AAV Gene Therapy in a Canine Model of MPS1 Prevents and Reverses Corneal Blindness
-
AAV Hijacks Cellular Secretion Machinery for Efficient Viral Egress
-
AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia.
-
AAV-CNS matters turn from gray to white.
-
AAV-Mediated Deletion of a Large Mutational Hotspot for Treatment of Duchenne Muscular Dystrophy
-
AAV-Mediated Gene Therapy Using a Novel Dual Promoter Prevents Pullulanase-Induced Cytotoxic T Lymphocyte Response and Corrects Major Affected Tissues in GSD IIIa Mice
-
AAV-Mediated Gene Therapy for Glycogen Storage Disease Type III Using a Bacterial Glycogen Debranching Enzyme
-
AAV-MediatedTAZ
Gene Replacement Restores Mitochondrial and Cardioskeletal Function in Barth Syndrome
-
AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.
-
APOE-Targeted Epigenome Therapy for Alzheimer's Disease
-
Activity of carbonic anhydrase immobilized on porous silica beads in organic media
-
Adeno-Associated Virus Capsid-Promoter Interactions in the Brain Translate from Rat to the Nonhuman Primate.
-
Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood.
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Adeno-associated viral vectors based on serotype 3b use components of the fibroblast growth factor receptor signaling complex for efficient transduction.
-
Adeno-associated virus serotypes: vector toolkit for human gene therapy.
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Adeno-associated virus-mediated gene therapy for metabolic myopathy.
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Adenovirus-mediated gene transfer of the beta2-adrenergic receptor to donor hearts enhances cardiac function.
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Adhesion mediated by bonds in series.
-
Adjunctive beta 2-Agonist Therapy Enhances Trafficking of Acid alpha-Glucosidase to Lysosomes in the Skeletal Muscle of Mice with Pompe Disease
-
Advances in our understanding of ligand-activated nuclear receptors
-
Advances in pharmacogenomic research and development.
-
Advances in quantitative UV-visible spectroscopy for clinical and pre-clinical application in cancer.
-
Advances in targeted genome editing.
-
Advancing islet transplantation: from engraftment to the immune response.
-
Allele Specific Knockdown of APOE e4 Expression: A Novel Platform for Precision Gene Therapy in Alzheimer's Disease
-
Alterations of endophytic microbial community function in Spartina alterniflora as a result of crude oil exposure.
-
Altered distribution of mitochondria and actin fibers in 3T3 cells cultured on microcarriers.
-
Altering Tissue Glycan Patterns Improves the Transduction Profile of AAV9 Vectors
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Amendment to Clinical Research Project. Project 90-C-195. April 1, 1993. Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with autologous lymphocytes transduced with a human ADA gene.
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An Endogenous TNF-α Antagonist Induced by Splice-switching Oligonucleotides Reduces Inflammation in Hepatitis and Arthritis Mouse Models.
-
An Isogenic hiPSC-Derived Model System for Establishing Gene Therapy Approaches in Alzheimer's Disease
-
An autonomously self-assembling dendritic DNA nanostructure for target DNA detection.
-
An efficient targeted radiotherapy/gene therapy strategy utilising human telomerase promoters and radioastatine and harnessing radiation-mediated bystander effects.
-
An emerging adeno-associated viral vector pipeline for cardiac gene therapy.
-
An endogenous TNF-alpha antagonist induced by splice-switching oligonucleotides reduces inflammation in hepatitis and arthritis mouse models.
-
An in vitro system for efficiently evaluating gene therapy approaches to hemoglobinopathies.
-
Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors.
-
Analysis of neuronal proliferation, migration and differentiation in the postnatal brain using equine infectious anemia virus-based lentiviral vectors.
-
Analysis of oxygen transport in a diffusion-limited model of engineered heart tissue.
-
Analysis of the rate-limiting step of an anaerobic biotrickling filter removing TCE vapors
-
Anti-cryptococcal activity of preussolides A and B, phosphoethanolamine-substituted 24-membered macrolides, and leptosin C from coprophilous isolates of Preussia typharum.
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Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease.
-
Antibody-antigen binding kinetics. A model for multivalency antibodies for large antigen systems.
-
Antidote control of aptamer therapeutics: the road to a safer class of drug agents.
-
Antisettlement and Narcotic Action of Analogues of Diterpene Marine Natural Product Antifoulants from Octocorals.
-
Antiviral Effect of Retro-2.1 against Herpes Simplex Virus Type 2 In Vitro.
-
Appearance of protease activities coincides with p10 and polyhedrin-driven protein production in the baculovirus expression system: effects on yield.
-
Application of a moving bed biofilm reactor for tertiary ammonia treatment in high temperature industrial wastewater.
-
Application of an antibody biochip for p53 detection and cancer diagnosis.
-
Applications of nanotechnology to biotechnology commentary.
-
Approaches to HIV vaccine design.
-
Aptamer-siRNA Chimera Inhibitors of Intimal Hyperplasia
-
Aptamers in immunotherapy.
-
Aquaporins and CSF Flux Are Critical Determinants of AAV Mediated CNS Gene Transfer
-
B-cell-lineage immunogen design in vaccine development with HIV-1 as a case study.
-
Bacterial removal of mercury from sewage.
-
Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model.
-
Bezafibrate Enhances AAV Gene Therapy in Glycogen Storage Disease Type Ia
-
Bimodular CRISPR/Cas-lentiviral Vector for Modeling Diseases
-
Bioactive poly(ethylene glycol) hydrogels to recapitulate the HSC niche and facilitate HSC expansion in culture.
-
Biocatalytic coatings for air pollution control: a proof of concept study on VOC biodegradation.
-
Biochemical, morphological, and genetic variations in Microcystis aeruginosa due to colony disaggregation
-
Bioengineered Skeletal Muscle With Functional Stem Cell Pool and Capacity for Vascular Integration and Maturation In Vivo
-
Biological function made crystal clear - annotation of hypothetical proteins via structural genomics.
-
Biological waste air treatment in biofilters.
-
Biological waste air treatment in biotrickling filters
-
Biology by design: reduction and synthesis of cellular components and behaviour.
-
Bioluminescent virion shells: new tools for quantitation of AAV vector dynamics in cells and live animals.
-
Biomass control in waste air biotrickling filters by protozoan predation.
-
Biomaterializing the promise of cardiac tissue engineering.
-
Biomechanics of vascular-stent interaction and in-stent restenosis: The key role of shear stress
-
Biomedical implications of protein folding and misfolding.
-
Biotreatment of groundwater contaminated with MTBE: interaction of common environmental co-contaminants.
-
Boosting, Not Breaking: CRISPR Activators Treat Disease Models.
-
Breast cancer: integrating the patient with her genome.
-
CRISPR-Mediated Gene Correction in a Severe Humanized Mouse Model of Duchenne Muscular Dystrophy
-
CRISPR-based methods for high-throughput annotation of regulatory DNA.
-
CRISPR/Cas9 Inducible Systems for Neurodegenerative Disease
-
CRISPR/Cas9-Based Transcriptional Repressors for Control of Human Gene Expression
-
Capsid-Glycan Receptor Interactions Influence AAV Transport across the Blood-Brain Barrier
-
Cardiac I-1c overexpression with reengineered AAV improves cardiac function in swine ischemic heart failure.
-
Cardiac tissue engineering: cell seeding, cultivation parameters, and tissue construct characterization.
-
Catheter-based intracoronary myocardial adenoviral gene delivery: importance of intraluminal seal and infusion flow rate.
-
Catheter-mediated subselective intracoronary gene delivery to the rabbit heart: introduction of a novel method.
-
Cell population dynamics model for deconvolution of murine embryonic stem cell self-renewal and differentiation responses to cytokines and extracellular matrix.
-
Cell-Targeted RNA-Based Therapies for Cardiovascular Disease
-
Cellular arrays for large-scale analysis of transcription factor activity.
-
Cellular uptake of neutral phosphorodiamidate morpholino oligomers.
-
Centrifugation without a balance tube
-
Centromere round-up at the heterochromatin corral.
-
Characterization of Clostridium ljungdahlii OTA1: a non-autotrophic hyper ethanol-producing strain.
-
Characterization of Liver Pathology in a Novel GSD IX y2 Mouse Model
-
Characterization of bimodal cell death of insect cells in a rotating- wall vessel and shaker flask
-
Characterization of the distinct orthotopic bone-forming activity of 14 BMPs using recombinant adenovirus-mediated gene delivery.
-
Characterizing Essential Receptor Binding Residues for AAV1
-
Clinical applications of dendritic cell vaccines.
-
Co-opting biology to deliver drugs.
-
Colloidal gold as a biocompatible immobilization matrix suitable for the fabrication of enzyme electrodes by electrodeposition.
-
Colocalizing ribozymes with substrate RNAs to increase their efficacy as gene inhibitors.
-
Combination treatment of murine tumors by adenovirus-mediated local B7/IL12 immunotherapy and radiotherapy.
-
Comparative genomics as a tool for gene discovery.
-
Comparing genome editing technologies
-
Comparison of Gene Delivery to the Kidney by Adenovirus, Adeno-Associated Virus, and Lentiviral Vectors After Intravenous and Direct Kidney Injections.
-
Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria.
-
Computational Fluid Dynamics and Additive Manufacturing to Diagnose and Treat Cardiovascular Disease.
-
Computing. Successes and challenges.
-
Conspecific cues that induce spore settlement in the biofouling and green tide-forming alga Ulva tepida provide a potential aggregation mechanism
-
Continuous operation of foamed emulsion bioreactors treating toluene vapors.
-
Controlling cancer-induced inflammation with a nucleic acid scavenger prevents lung metastasis in murine models of breast cancer.
-
Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease.
-
Correction of Glycogen Storage Disease Type III with an AAV Vector Encoding a Bacterial Glycogen Debranching Enzyme
-
Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases
-
Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases.
-
Correction of glycogen storage disease type II (GSD II) by intramuscular administration of an adeno-associated virus (AAV) vector pseudotyped as AAV6
-
Correction of glycogen storage disease type II (GSD II) with an adeno-associated virus 8 (AAV2/8) vector
-
Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter.
-
Correction of glycogen storage disease type II by intramuscular administration of an adeno-associated virus 6 (AAV2/6) vector containing a muscle-specific promoter
-
Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy.
-
Corrective gene transfer into bone marrow CD34+cells for adenosine deaminase (ADA) deficiency: Results in four patients after one year of follow-up
-
Corrigendum to "Efficacy of Helper-dependent Adenovirus Vector-mediated Gene Therapy in Murine Glycogen Storage Disease Type Ia".
-
Critical assessment of gassing-in methods to determine mass transfer coefficient in miniature and microbioreactors with gas-liquid flow
-
Critical issues in delivery of RNAi therapeutics in vivo.
-
Cross-Species Evolution of Synthetic AAV Strains for Clinical Translation
-
Cultivation studies on wild and hybrid strains of Pleurotus tuberregium (Fr.) Sing. on wheat straw substrate.
-
Current technology for the derivation of pluripotent stem cell lines from human embryos.
-
Current trends in differential expression proteomics: isotopically coded tags.
-
D-amino acid oxidase and catalase of detergent permeabilized Rhodotorula gracilis cells and its potential use for the synthesis of α-keto acids
-
DNA patents and diagnostics: not a pretty picture.
-
DNA transfection of macaque and murine respiratory tissue is greatly enhanced by use of a nuclease inhibitor.
-
Decarbonizing agriculture through the conversion of animal manure to dietary protein and ammonia fertilizer.
-
Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to Adeno-Associated Virus Vector Containing a CMV Enhancer/Chicken beta-Actin Cassette in Murine Pompe Disease
-
Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy.
-
Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors.
-
Department of Health and Human Services National Institutes of Health Recombinant DNA Advisory Committee. Minutes of meeting March 8-10, 2000.
-
Depletion of Pre-Existing Anti-AAV Neutralizing Antibodies by a Combination Immunosuppressive Treatment with Bortezomib and CD20 mAb Allows Successful Vector Re-Administration in Mice
-
Desensitization through Immunomodulatory Gene Therapy Suppresses the Antibody Response to Enzyme Replacement Therapy in Murine Pompe Disease
-
Design Principles for AAV Mediated Circular RNA Expression in the Brain
-
Detailed investigations of dissolved hydrogen and hydrogen mass transfer in a biotrickling filter for upgrading biogas.
-
Developing elite Neurospora crassa strains for cellulosic ethanol production using fungal breeding.
-
Development of AAV-Based CRISPR/Cas9 Therapies for Correcting Duchenne Muscular Dystrophy by Targeted Genomic Integration
-
Development of AAV-shRNA vectors for human basal-like breast cancer therapy
-
Development of Lentiviral Platform for Allele-Specific Targeted Down-Regulation of APOEe4 Expression: Epigenetic Therapy for Precision Medicine in Alzheimer's Disease
-
Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer.
-
Development of a novel intravascular oxygenator catheter: Oxygen mass transfer properties across nonporous hollow fiber membranes.
-
Development of adeno-associated virus (AAV) vectors for gene therapy in the mouse model for glycogen storage disease type Ia (GSD-Ia)
-
Development of foamed emulsion bioreactor for air pollution control.
-
Development of mammalian serum albumin affinity purification media by peptide phage display.
-
Development of novel biotherapeutics for breast cancer: generation of function blocking antibodies against Notch1 ligands
-
Diagnosis of fungal infection: new technologies for the mycology laboratory.
-
Diamagnetic levitation changes growth, cell cycle, and gene expression of Saccharomyces cerevisiae.
-
Differential Induction of Immunogenic Cell Death and Interferon Expression in Cancer Cells by Structured ssRNAs.
-
Digital image analysis for rapid quantification of total RNA and cDNA for SMART-PCR.
-
Digital pathology in nephrology clinical trials, research, and pathology practice.
-
Direct Comparison of Epifluorescence and Immunostaining for Assessing Viral Mediated Gene Expression in the Primate Brain.
-
Direct force measurements of the streptavidin-biotin interaction.
-
Directing three-dimensional multicellular morphogenesis by self-organization of vascular mesenchymal cells in hyaluronic acid hydrogels
-
Discontinuation of PEG-ADA replacement therapy in a patient with ADA-deficiency previously treated with retroviral-mediated, T cell-directed gene therapy
-
Discovery of a Neurotropic Footprint That Enables AAV Transport Across the Blood-Brain Barrier
-
Disruption of Water Homeostasis Affects AAV Transport Across the Blood-Brain Barrier
-
Distinct Functional Roles for AAV Capsid Surface Epitopes Revealed through Structure-Guided Evolution
-
Downregulation of SNCA Expression as a Therapoetic Approach for Parkinson's Disease and Other Synucleinopathies: New Target Validation for Next-Generation Drug Discovery
-
Downregulation of SNCA Expression by Targeted Editing of DNA Methylation: A Potential Strategy for Precision Therapy in PD.
-
Drag reducing properties of microalgal exopolymers.
-
Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α.
-
Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. Aureus Cas9
-
Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. aureus Cas9
-
EZH2 RIP-seq Identifies Tissue-specific Long Non-coding RNAs.
-
Early marine bacterial biofilm on a copper-based antifouling paint
-
Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia.
-
Editing out Five SERPINA1 Paralogs to Create a New Mouse Model of Genetic Emphysema
-
Editorial overview - A cornucopia of strategies for the immunotherapy of cancer
-
Editorial overview - A need for effective adjuvants
-
Editorial overview: Fulfilling the promise of immunotherapy
-
Editorial overview: Priming, boosting, measuring
-
Editorial overview: What is a vaccine?
-
Effect of AAV Gene Therapy on Hepatic Tumor Induced by Deficiency of Glucose-6-Phosphatase
-
Effect of carbon source addition on toluene biodegradation by an Escherichia coli DH5alpha transconjugant harboring the TOL plasmid.
-
Effect of genetic circular permutation near the active site on the activity and stability of an enzyme inhibitor.
-
Effect of streptavidin RGD mutant on the adhesion of endothelial cells.
-
Effects of epidermal growth factor on fibroblast migration through biomimetic hydrogels.
-
Effects of medical research on health care and economy.
-
Effects of unsteady state conditions on the biooxidation of methyl ethyl and methyl isobutyl ketone in continuous flow liquid phase cultures
-
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease.
-
Efficacy of Genome Editing in Infant Mice with Glycogen Storage Disease Type Ia
-
Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.
-
Efficacy of gene therapy for a prototypical lysosomal storage disease (GSD-II) is critically dependent on vector dose, transgene promoter, and the tissues targeted for vector transduction.
-
Efficacy of helper-dependent adenovirus vector-mediated gene therapy in murine glycogen storage disease type Ia.
-
Efficacy of multiple administrations of a recombinant adenovirus expressing wild-type p53 in an immune-competent mouse tumor model.
-
Electromobility of plasmid DNA in tumor tissues during electric field-mediated gene delivery.
-
Electron capture dissociation mass spectrometry in characterization of peptides and proteins.
-
Emerging strategies for engineering microbial communities.
-
Enabling Technologies for Personalized and Precision Medicine.
-
Endophytic Phytoaugmentation: Treating Wastewater and Runoff Through Augmented Phytoremediation.
-
Energy biotechnology in 2013: advanced technology development for breakthroughs in fuels and chemicals production.
-
Engineered cell-cell communication and its applications.
-
Engineered microbial consortia: strategies and applications.
-
Engineered pluripotent mesenchymal cells integrate and differentiate in regenerating bone: a novel cell-mediated gene therapy.
-
Engineered pluripotent mesenchymal cells integrate and differentiate in regenerating bone: a novel cell-mediated gene therapy.
-
Engineered transcription factors for therapeutic angiogenesis.
-
Engineering A Humanized AAV8 Capsid through Iterative Structure-Guided Evolution
-
Engineering Backsplicing Introns for Robust Expression of Synthetic Circular RNAs from AAV Vectors
-
Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles.
-
Engineering clustered ligand binding into nonviral vectors: αvβ3 targeting as an example
-
Engineering life: building a fab for biology.
-
Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer.
-
Engineering microbial consortia: a new frontier in synthetic biology.
-
Engineering microbial systems to explore ecological and evolutionary dynamics.
-
Engineering multicellular systems by cell-cell communication.
-
Engineering skeletal muscle repair.
-
Engineering superficial zone features in tissue engineered cartilage.
-
Enhanced AAV vector transduction through genetic and pharmacological inhibition of U2 snRNP components
-
Enhanced CRISPR/Cas9 Genome Editing in Heart and Skeletal Muscle with a Potent New AAV Variant
-
Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade.
-
Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.
-
Enhanced oncolytic virotherapy through oxidative stress inhibition.
-
Enhanced pancreatic cancer gene therapy by combination of adenoviral vector expressing c-erb-B2 (Her-2/neu)-targeted immunotoxin with a replication-competent adenovirus or etoposide.
-
Enhancement of targeted radiotherapy in neuroblastoma: a novel gene therapy approach
-
Enhancing RNA repair efficiency by combining trans-splicing ribozymes that recognize different accessible sites on a target RNA.
-
Enhancing immunogenicity by CpG DNA.
-
Enzymatic RNA molecules
-
Enzymatic deglycosylation of enfumafungin, a triterpene glycoside natural product, and its chemically synthesized analogues
-
Epigenetic Downregulation of ApoE4 for Alzheimer's Disease
-
Epigenome-Base Strategies for Efficient and Safe Editing of APOE epsilon 4 Allele: Implications for Alzheimer's Disease
-
Erratum to Analysis of the rate-limiting step of an anaerobic biotrickling filter removing TCE vapors, [Process Biochem. (2010), 45, 549-555]
-
Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia (Gene Therapy (2006) vol. 13 (1281-1289) 10.1038/sj.gt.3302774)
-
Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia (Gene Therapy (2006) vol. 13 (1281-1289) 10.1038/sj.gt.3302774))
-
Erratum: Reading Frame correction by targeted genome editing restores dystrophin expression in cells from duchenne muscular dystrophy patients (Molecular Therapy (2013) 21 (1718-1726) DOI: 10.1038/mt.2013.111)
-
Ethics for the pediatrician: a brave new pediatrics? Managing the desire for better children through biotechnological enhancement.
-
Evaluating the mycostimulation potential of select carbon amendments for the degradation of a model PAH by an ascomycete strain enriched from a superfund site.
-
Evaluating whole transcriptome amplification for gene profiling experiments using RNA-Seq
-
Evaluation of repRNA vaccine for induction and in utero transfer of maternal antibodies in a pregnant rabbit model.
-
Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.
-
Evolution of a New AAV Variant with Murine T Lymphocyte Tropism Using the MHC-Ib Molecule H2-Q7 as a Receptor
-
Evolving New AAV Strains that Demonstrate AAVR Independence
-
Evolving Synthetic AAV Variants for Genome Editing in Immune Cell Populations
-
Ex Vivo Gene Delivery to Porcine Cardiac Allografts Using a Myocardial-Enhanced Adeno-Associated Viral Vector.
-
Examining the behavior of crop-derived antibiotic resistance genes in anaerobic sludge batch reactors under thermophilic conditions.
-
Exercise and health: can biotechnology confer similar benefits?
-
Expanding the CNS Transduction Profile of AAV4 by Structure-Guided Evolution
-
Exploring Novel Aptamer-Heparin Combinations to Improve Anticoagulation Therapy in Cardiopulmonary Bypass
-
Expression of Naked Plasmid DNA Injected into the Afferent and Efferent Vessels of Rodent and Dog Livers
-
FINDING TARGETS FOR FSH DYSTROPHY THERAPY
-
Fibrillar peptide gels in biotechnology and biomedicine.
-
Flow cytometry analysis of adenosine deaminase (ADA) expression: a simple and reliable tool for the assessment of ADA-deficient patients before and after gene therapy.
-
Focused ultrasound (HIFU) induces localized enhancement of reporter gene expression in rabbit carotid artery.
-
Formulations which increase the size of lipoplexes prevent serum-associated inhibition of transfection.
-
From crystal structure to clinic: highlights of the Tenth International Parvovirus Workshop.
-
From the editor: Trust and the value paradox in science.
-
Fulfilling the promise of immunotherapy.
-
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.
-
Functional advantages conferred by extracellular prokaryotic membrane vesicles.
-
Functionality of the TOL plasmid under varying environmental conditions following conjugal transfer.
-
Furin is a Host Factor Restricting Adeno-Associated Virus 4 Transduction
-
G protein-coupled receptors and receptor kinases: from molecular biology to potential therapeutic applications.
-
GENIS: gene expression of sodium iodide symporter for noninvasive imaging of gene therapy vectors and quantification of gene expression in vivo.
-
Gene Therapy for Heart Disease Using Electrically Active Fibroblasts
-
Gene Therapy for Modulation of T-Cell-Mediated Immune Response Provoked by Corneal Transplantation.
-
Gene delivery through cell culture substrate adsorbed DNA complexes
-
Gene expression and survival changes in Saccharomyces cerevisiae during suspension culture.
-
Gene expression profiling of anticancer immune responses.
-
Gene therapy for cardiovascular disease.
-
Gene therapy progress and prospects: RNA aptamers.
-
Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.
-
Generation of recombinant adeno-associated virus vectors by a complete adenovirus-mediated approach.
-
Generation of species cross-reactive aptamers using "toggle" SELEX.
-
Genetic engineering of mesenchymal stem cells and its application in human disease therapy.
-
Genetic profiling and tailored therapy in asthma: are we there yet?
-
Genetic studies: look no further than your own backyard.
-
Genetically encoded elastin-like polypeptide nanoparticles for drug delivery.
-
Genome Editing in a Canine Model for Glycogen Storage Disease Type Ia
-
Genome-editing Technologies for Gene and Cell Therapy.
-
Genomics-based anti-cancer drug discovery - Preface
-
Glyco-engineering in Archaea: differential N-glycosylation of the S-layer glycoprotein in a transformed Haloferax volcanii strain.
-
Glycogen storage in multiple muscles of old GSD-II mice can be rapidly cleared after a single intravenous injection with a modified adenoviral vector expressing hGAA.
-
Granular hydrogels: emergent properties of jammed hydrogel microparticles and their applications in tissue repair and regeneration.
-
Grazing scar characteristics impact degree of fungal facilitation in spartina alterniflora leaves in a south american salt marsh
-
Group I intron-mediated trans-splicing revision of mutant p53 RNA transcripts
-
HSV oncolytic therapy upregulates interferon-inducible chemokines and recruits immune effector cells in ovarian cancer.
-
HSV-1 amplicon vector-mediated expression of ATM cDNA and correction of the ataxia-telangiectasia cellular phenotype.
-
Health care costs.
-
Hematopoietic Stem Cell Gene Therapy with Lentiviral Vector in 4 Patients with Cerebral X-Linked Adrenoleukodystrophy: Long-Term Outcome and Comparison of Efficacy with Allogeneic Hematopoietic Stem Cell Transplantation
-
Hematopoietic stem cell gene therapy with lentiviral vector in 4 patients with cerebral X-linked adrenoleukodystrophy: long-term outcome and comparison of efficacy with allogeneic hematopoietic stem cell transplantation
-
Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector.
-
High-Performance Biogas Upgrading Using a Biotrickling Filter and Hydrogenotrophic Methanogens.
-
High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline.
-
High-throughput phenotyping of multicellular organisms: finding the link between genotype and phenotype.
-
Homozygous diploid deletion strains of Saccharomyces cerevisiae that determine lag phase and dehydration tolerance.
-
Host Transcription Factors and Co-Opted Signaling Pathways Orchestrate Epigenetic Modulation of the AAV Vector Genome
-
How changes in drug-safety regulations affect the way drug and biotech companies invest in innovation.
-
How eco-evolutionary principles can guide tree breeding and tree biotechnology for enhanced productivity.
-
Human Pluripotent Stem Cell-Derived Cardiac Tissue Patch for Use in Cell-Based Cardiac Therapy
-
Human carbonic anhydrase-8 AAV8 gene therapy inhibits nerve growth factor signaling producing prolonged analgesia and anti-hyperalgesia in mice.
-
Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6
-
Human interleukin-2 production in insect (Trichoplusia ni) larvae: effects and partial control of proteolysis.
-
Hydrogels for brain repair after stroke: an emerging treatment option
-
Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease.
-
Hypoxic cell death is reduced by pH buffering in a model of engineered heart tissue.
-
Identification of a homogentisate-1,2-dioxygenase gene in the fungus Exophiala lecanii-corni: analysis and implications.
-
Identification of cyclosporin C from Amphichorda felina using a Cryptococcus neoformans differential temperature sensitivity assay.
-
Identification of fungi associated with municipal compost using DNA-based techniques.
-
Identifying bioaugmentation candidates for bioremediation of polycyclic aromatic hydrocarbons in contaminated estuarine sediment of the Elizabeth River, VA, USA.
-
Imaging Biomaterial-Tissue Interactions.
-
Imaging Trafficking Dynamics of Ultra-Pure Single Adeno-Associated Virus in Live Cells
-
Imaging of cell/substrate contacts on polymers by total internal reflection fluorescence microscopy.
-
Immune Reconstitution after Gene Therapy for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID)
-
Immunity to Cas9 as an Obstacle to Persistent Genome Editing.
-
Immunization with an SIV-based IDLV Expressing HIV-1 Env 1086 Clade C Elicits Durable Humoral and Cellular Responses in Rhesus Macaques.
-
Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease.
-
Immunomodulatory gene therapy in lysosomal storage disorders.
-
Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.
-
Immunotherapeutic targeting of Wilms' tumor protein.
-
Immunotherapy of cancer with dendritic cell-based vaccines.
-
Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression.
-
Implications of Gene Therapy for Alzheimer's
-
Important cellular targets for antimicrobial photodynamic therapy.
-
Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model.
-
Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice.
-
In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation.
-
In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation.
-
In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.
-
In vivo reprogramming of hTERT by trans-splicing ribozyme to target tumor cells.
-
In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using non-primate lentiviral vectors
-
Increased Frequency of Permanent Integration Following Liver-Directed Gene Therapy with rDNA-AAV2/8 In Murine Phenylketonuria
-
Increasing the specificity of CRISPR systems with engineered RNA secondary structures.
-
Inducible regulation of Runx2-stimulated osteogenesis.
-
Inhalation of an RNA Aptamer Targeting Extracellular Histones Protects from Acute Lung Injury
-
Inhibition of Caspase-3 Improves Electrotransfer Efficiency and Cell Viability
-
Inhibition of Extracellular Histones in Sepsis-Induced Cardiovascular Function
-
Inhibition of barnacle larval settlement and crustacean toxicity of some hoplonemertine pyridyl alkaloids.
-
Inhibitory activity of probiotics Streptococcus phocae PI80 and Enterococcus faecium MC13 against Vibriosis in shrimp Penaeus monodon
-
Innovative bioreactors.
-
Insertional analyses in rhesus monkey blood cells after non-myeloablative hematopoietic stem cell marking with a therapeutic onco-retroviral vector for X-linked chronic granulomatous disease
-
Integrase-Deficient Lentiviral Vector for Efficient CRISPR/Cas9-Mediated Gene and Epigenome-Editing Applications
-
Integration of cell-free protein coexpression with an enzyme-linked immunosorbent assay enables rapid analysis of protein-protein interactions directly from DNA.
-
Integrins Play a Pivotal Role in Transvascular Transport and Tissue Uptake of AAV9 In Vivo
-
Intra-arterial administration of a replication-selective adenovirus (dl1520) in patients with colorectal carcinoma metastatic to the liver: a phase I trial.
-
Intralingual Administration of AAVrh10-miRSOD1 Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis.
-
Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease.
-
Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I.
-
Investigation of cell kill in a targeted radiotherapy/gene therapy strategy utilising radiation induced biological bystander effects
-
Involvement of a Rac1-Dependent Macropinocytosis Pathway in Plasmid DNA Delivery by Electrotransfection.
-
In Vivo Expansion and Antitumor Activity of Coinfused CD28- and 4-1BB-Engineered CAR-T Cells in Patients with B Cell Leukemia.
-
Ion channel engineering for modulation and de novo generation of electrical excitability.
-
Isolation and insecticidal activity of mellamide from Aspergillus melleus.
-
Isolation of suppressor mutants of phosphatidylinositol 3-phosphate 5-kinase deficient cells in Schizosaccharomyces pombe.
-
Isolation, growth and differentiation of adult rabbit skeletal myoblasts in vitro
-
Isolation, structure, and HIV-1-integrase inhibitory activity of structurally diverse fungal metabolites.
-
It's All in the Delivery: Designing Hydrogels for Cell and Non-viral Gene Therapies.
-
Keeping Sample Preparation Simple: Tips on How to Perform Proteomics Measurements with Consistency and Accuracy
-
Kinetic studies and unstructured models of lymphocyte metabolism in fed-batch culture.
-
Large scale purification of factor X by hydrophobic chromatography.
-
Laser-scanning lithography (LSL) for the soft lithographic patterning of cell-adhesive self-assembled monolayers.
-
Laser-scanning velocimetry: a confocal microscopy method for quantitative measurement of cardiovascular performance in zebrafish embryos and larvae.
-
Learning from each other: Applying observations from anti-tumor, anti-pathogen and anti-toxin immunotherapy
-
Lentiviral gene transduction of kidney.
-
Letter to the Editor: mRNA Vaccines in Urological Malignancies.
-
Lipid-based Vehicles for siRNA Delivery in Biomedical Field.
-
Liver Directed AAV Gene Therapy Reverses Progression of Glycogen Storage Disease Type IX gamma 2 in Mice
-
Liver-Mediated Gene Therapy with an Engineered Secretable GAA Transgene Results in Whole-Body Correction of Pompe Disease
-
Long-Term Characterization of In Vivo Genome Editing in a Mouse Model of Duchenne Muscular Dystrophy
-
Long-Term Correction of Glycogen Storage Disease Type III by AAV-Mediated Gene Therapy Using a Dual Promoter to Express Bacterial Pullulanase
-
Long-Term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy
-
Long-Term Evaluation of Genome Editing Outcomes for Duchenne Muscular Dystrophy
-
Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector.
-
Long-term efficacy after [E1-, polymerase-] adenovirus-mediated transfer of human acid-alpha-glucosidase gene into glycogen storage disease type II knockout mice.
-
Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia.
-
Long-term follow-up assessment of a phase 1 trial of angiogenic gene therapy using direct intramyocardial administration of an adenoviral vector expressing the VEGF121 cDNA for the treatment of diffuse coronary artery disease.
-
Loperamide-based compounds as additives for biofouling management
-
Lymphatic Transport Mediates Systemic Leakage of Peripherally Administered AAV9 Vectors
-
MAPK signal-integrating kinase controls cap-independent translation and cell type-specific cytotoxicity of an oncolytic poliovirus.
-
Macrophage colony-stimulating factor can modulate immune responses and attract dendritic cells in vivo.
-
Mapping and Engineering Functional Domains of the Assembly Activating Protein of Adeno-Associated Viruses
-
Mapping the Antigenic Structure of Adeno-Associated Virus Serotypes 8 and 9
-
Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier.
-
Mass Spectrometric Identification of Host Factors that Restrict AAV Vector Genome Transcription
-
Matrix-based gene delivery for tissue repair
-
Mechanism and inhibition of LpxC: an essential zinc-dependent deacetylase of bacterial lipid A synthesis.
-
Mechanism of Shortened Bones in Mucopolysaccharidosis VII
-
Mechanistic Elucidation of Adenovirus Mediated Enhancement of Recombinant Adeno-Associated Virus (rAAV) towards Efficient Low Dose Gene Therapy
-
Mediator-free amperometric determination of toxic substances based on their inhibition of immobilized horseradish peroxidase.
-
Medical technologies for the diagnosis of prostate cancer.
-
Medicine. The need for a global HIV vaccine enterprise.
-
Mesenchymal stem cells overexpressing Akt dramatically repair infarcted myocardium and improve cardiac function despite infrequent cellular fusion or differentiation.
-
Metabolic engineering of Escherichia coli to produce a monophosphoryl lipid A adjuvant.
-
Metabolic regulation of epigenetic remodeling in immune cells.
-
Metabolomics applied to diabetes research: moving from information to knowledge.
-
Metalloprotein design.
-
Methotrexate and cytarabine inhibit progression of human lymphoma in NOD/SCID mice carrying a mutant dihydrofolate reductase and cytidine deaminase fusion gene.
-
Microparticles bearing encephalitogenic peptides induce T-cell tolerance and ameliorate experimental autoimmune encephalomyelitis.
-
Milligram scale production of potent recombinant small interfering RNAs in Escherichia coli.
-
Minimum Effective Dose to Achieve Biochemical Correction with Adeno-Associated Virus Vector-Mediated Gene Therapy in Mice with Pompe Disease.
-
Modeling of a foamed emulsion bioreactor: I. Model development and experimental validation.
-
Modeling of a foamed emulsion bioreactor: II. model parametric sensitivity.
-
Modular nanotransporters: a multipurpose in vivo working platform for targeted drug delivery.
-
Mold-casted non-degradable, islet macro-encapsulating hydrogel devices for restoration of normoglycemia in diabetic mice.
-
Molecular cloning and recombinant expression of a gene encoding a fungal immunomodulatory protein from Ganoderma lucidum in Pichia pastoris
-
Monitoring cellular immune responses to cancer immunotherapy.
-
Multiple muscles in the AMD quail can be "cross-corrected" of pathologic glycogen accumulation after intravenous injection of an [E1-, polymerase-] adenovirus vector encoding human acid-alpha-glucosidase.
-
Mutation Detection Following Non-Homologous End Joining (NHEJ): A Comparison of Different Semi Quantitative and Quantitative Methods
-
Myocardial injury-induced fibroblast proliferation facilitates retroviral-mediated gene transfer to the rat heart in vivo.
-
Naked DNA delivered intraportally expresses efficiently in hepatocytes.
-
Nanopatterned polymer brushes: conformation, fabrication and applications.
-
Nanotechnology-based strategies for combating toxicity and resistance in melanoma therapy.
-
Natural killer cell activation and dendritic cell-based vaccines
-
Neonatal rAAV2/8 Vector Administration Does Not Prevent Therapeutic Response To Repeat Vector Injection in Adult Pah(enu2/enu2) Mice
-
Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia.
-
Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics.
-
Non-canonical role of Hippo tumor suppressor serine/threonine kinase 3 STK3 in prostate cancer.
-
Notable reduction in illegitimate integration mediated by a PPT-deleted, nonintegrating lentiviral vector
-
Novel Gene Supplementation, Genome Editing and Cellular Therapeutic Approaches to Treat Glutaric Aciduria Type I
-
Novel encapsulation of partially dehydrated protein microparticles in PLGA microspheres
-
Novel integrase-defective lentiviral episomal vectors for gene transfer.
-
Nuclear hormone receptors as targets for new drug discovery.
-
Nucleic acid aptamers as adjuncts to vaccine development.
-
Nucleic acid aptamers in therapeutic anticoagulation. Technology, development and clinical application.
-
Nucleic-Acid Binding Polymers as Anti-Inflammatory Agents
-
On the road to effective vaccine development
-
Oncolytic HSV exerts direct antiangiogenic activity in ovarian carcinoma.
-
Oncolytic poliovirus recombinants for the treatment of malignant glioma
-
Optical nanosensors and biological measurements
-
Optimization of in vivo crosslinking technique for the study of AlpB-protein interactions in Lysobacter sp. XL1 cells
-
Optimizing aptamer activity for gene therapy applications using expression cassette SELEX.
-
Overview of CT technologies for children.
-
Oxypropylation of cork residues: Preliminary results
-
PEGDA hydrogels with patterned elasticity: Novel tools for the study of cell response to substrate rigidity.
-
Packaging of an AAV vector encoding human acid alpha-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector.
-
Pain assessment and treatment disparities: a virtual human technology investigation.
-
Pathogenesis of Growth Failure in Murine and Canine Glycogen Storage Disease-Ia and Partial Reversal of Growth Failure Using Adeno-Associated Viral Vector Therapy
-
Patient-specific iPSC-derived endothelial cells provide longterm phenotypic correction of hemophilia A
-
Patterns of regulation from mRNA and protein time series.
-
Peptide affinity reagents for AAV capsid recognition and purification.
-
Performance evaluation of a full-scale innovative swine waste-to-energy system.
-
Performance of Homologous and Heterologous Prime-Boost Immunization Regimens of Recombinant Adenovirus and Modified Vaccinia Virus Ankara Expressing an Ag85B-TB10.4 Fusion Protein against Mycobacterium tuberculosis.
-
Persistent, therapeutically relevant levels of human granulocyte colony-stimulating factor in mice after systemic delivery of adeno-associated virus vectors.
-
Personalized diagnostics and biosensors: a review of the biology and technology needed for personalized medicine.
-
Personalized health care: from theory to practice.
-
Personalized medicine: revolutionizing drug discovery and patient care.
-
Perspectives on the development of anti-HIV vaccines.
-
Pharmacological regulation of dendritic cell activation: Enhancing cancer vaccine efficacy
-
Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Analyses of Safety and Secondary Endpoints
-
Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Initial 104-Week Experience
-
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.
-
Phase I study of liver depot gene therapy in late-onset Pompe disease.
-
Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID)
-
Physical Positioning Dramatically Improves Brain Transduction after Intrathecal Infusion of AAV9
-
Picoliter DNA sequencing chemistry on an electrowetting-based digital microfluidic platform.
-
Plant stomata: a checkpoint of host immunity and pathogen virulence.
-
Plant-centered biosystems in space environments: technological concepts for developing a plant genetic assessment and control system.
-
Policies and Pitfalls: What the Alzheimer's Community Can Learn from the Deployment of Disease-Modifying Therapies for Multiple Sclerosis Patients
-
Polyclonal long-term MFGS-gp91phox marking in rhesus macaques after nonmyeloablative transplantation with transduced autologous peripheral blood progenitor cells.
-
Polymer-Mediated Inhibition of Pro-invasive Nucleic Acid DAMPs and Microvesicles Limits Pancreatic Cancer Metastasis.
-
Potentially functional genetic variants of VAV2 and PSMA4 in the immune-activation pathway and non-small cell lung cancer survival.
-
Potentially functional genetic variants of the notch signaling pathway genes predict survival of Chinese patients with esophageal squamous cell carcinoma.
-
Powerful ideas driven by simple tools: lessons from experimental embryology.
-
Pre-Existing Humoral Immunity to AAV Capsids: Species and Age-Related Differences in Anti-Capsid ELISA and Neutralizing Antibody Assays
-
Pre-emptive gene therapy using recombinant adeno-associated virus delivery of extracellular superoxide dismutase protects heart against ischemic reperfusion injury, improves ventricular function and prolongs survival.
-
Preclinical Development of New Therapy for Glycogen Storage Diseases.
-
Preclinical Development of a vWF Aptamer to Limit Thrombosis and Engender Arterial Recanalization of Occluded Vessels.
-
Predation of bacteria by the protozoa Tetrahymena pyriformis in toluene-degrading cultures
-
Preface
-
Prenatal gene tranfer: scientific, medical, and ethical issues: a report of the Recombinant DNA Advisory Committee.
-
Preparation and activity of carbonic anhydrase immobilized on porous silica beads and graphite rods.
-
Preparation of peptide-loaded dendritic cells for cancer immunotherapy.
-
Prevalence and patterns of Transmitted Drug Resistance in HIV-Infected Adult Patients Initiating Antiretroviral Therapy in Hanoi, Vietnam
-
Prevalence of AAV1 neutralizing antibodies and consequences for a clinical trial of gene transfer for advanced heart failure.
-
Priming, boosting, measuring.
-
Production and quality control assessment of a GLP-grade immunotoxin, D2C7-(scdsFv)-PE38KDEL, for a phase I/II clinical trial.
-
Programming microbial population dynamics by engineered cell-cell communication.
-
Properties affecting transfer and expression of degradative plasmids for the purpose of bioremediation.
-
Prostate cancer: status of current treatments and emerging antisense-based therapies.
-
Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: A strategy for broad clinical application
-
Protein engineering and the development of generic biosensors.
-
Protein engineering approaches to biomaterials design.
-
Protein patterning.
-
Protein purification by fusion with an environmentally responsive elastin-like polypeptide: effect of polypeptide length on the purification of thioredoxin.
-
Proteolytically degradable hydrogels with a fluorogenic substrate for studies of cellular proteolytic activity and migration.
-
Proteomics for monitoring immune responses to cancer vaccines.
-
Proximity labeling of endogenous protein interactions enabled by directed evolution.
-
Purification of an elastin-like fusion protein by microfiltration.
-
Quantitation of cell area on glass and fibronectin-coated surfaces by digital image analysis.
-
REG-1, a regimen comprising RB-006, a Factor IXa antagonist, and its oligonucleotide active control agent RB-007 for the potential treatment of arterial thrombosis.
-
RNA Aptamer-targeted Inhibition of NF-κB Suppresses Non-small Cell Lung Cancer Resistance to Doxorubicin.
-
RNA Transfected Dendritic Cell Vaccines Targeting Human Cytomegalovirus Antigens in Patients with Glioblastoma
-
RNA aptamer blockade of osteopontin inhibits growth and metastasis of MDA-MB231 breast cancer cells.
-
RNA repair as a novel approach to genetic therapy.
-
RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
-
RNA transfected dendritic cells as cancer vaccines.
-
RNA-Modified T Cells Mediate Effective Delivery of Immunomodulatory Cytokines to Brain Tumors.
-
RNF121 is a Key Transcriptional Regulator of AAV Genome Expression
-
Rapidly regulating platelet activity in vivo with an antidote controlled platelet inhibitor.
-
Re-Engineering Capsid-Glycan Interactions Expands the CNS Transduction Profile of AAV Serotype 4
-
Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients.
-
Reading frame correction by targeted genome editing restores dystrophin expression in cells from duchenne muscular dystrophy patients
-
Recognition of glioma stem cells by genetically modified T cells targeting EGFRvIII and development of adoptive cell therapy for glioma.
-
Recombinant DNA Advisory Committee. Department of Health and Human Services. National Institutes of Health. Minutes of meeting. December 15-16, 1997.
-
Recombinant DNA Advisory Committee. Department of Health and Human Services. National Institutes of Health. Minutes of meeting. March 10, 1998.
-
Recombinant oncolytic poliovirus eliminates glioma in vivo without genetic adaptation to a pathogenic phenotype.
-
Recovery of pyruvic acid from biotransformation solutions.
-
Recruitment of bloom-forming cyanobacteria and its driving factors
-
Redirecting migration of T cells to chemokine secreted from tumors by genetic modification with CXCR2.
-
Reduction of Autophagic Accumulation in Pompe Disease Mouse Model Following Gene Therapy.
-
Reduction of invasive bacteria in ethanol fermentations using bacteriophages.
-
Regional-CNS MiniPromoters for AAV Are Identified in a High Through-Put Pipeline
-
Regulation of plant root system architecture: implications for crop advancement.
-
Relative gene expression quantification in a fungal gas-phase biofilter.
-
Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing.
-
Reporting Long Term Survival Following Precision Tumor-Targeted Gene Delivery to Advanced Chemotherapy-Resistant Malignancies: An Academic Milestone
-
Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia.
-
Resistance of pancreatic carcinoma cells is reversed by coculturing NK-like T cells with dendritic cells pulsed with tumor-derived RNA and CA 19-9.
-
Respiratory Directed Gene Therapy to Silence SOD1 Prolongs Survival in the SOD1 ALS Mouse
-
Response to Goldman and Brown: Making sense of microbial consortia using ecology and evolution
-
Responsible Use of Human Gene-Editing Technologies.
-
Results of One Year Follow-Up After Treatment With Fordadistrogene Movaparvovec (PF-06939926) for Duchenne Muscular Dystrophy (DMD) in A Phase 1b, Open-label Study
-
Retargeting mobile group II introns to repair mutant genes.
-
Retroviral delivery of GAD-IgG fusion construct induces tolerance and modulates diabetes: a role for CD4+ regulatory T cells and TGF-β?
-
Review: Tissue engineering in the nervous system
-
Ribozyme-mediated induction of apoptosis in human cancer cells by targeted repair of mutant p53 RNA.
-
Rich before old?
-
Rna Inhibitors of Nuclear Proteins Implicated in Multiple Organ Dysfunction Syndrome
-
Role of Vp2 in AAV packaging
-
Role of pericellular matrix in development of a mechanically functional neocartilage.
-
Runx2/Cbfa1-genetically engineered skeletal myoblasts mineralize collagen scaffolds in vitro.
-
STRV5, a Liver Detargeted AAV Capsid Demonstrates Improved Safety Profile After Intravenous Administration of High Doses in Non-Human Primates
-
Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development.
-
Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy.
-
Salmeterol with Liver Depot Gene Therapy Enhances the Skeletal Muscle Response in Murine Pompe Disease.
-
Salmeterol with Liver Depot Gene Therapy Reversed Biochemical and Autophagic Abnormalities of Skeletal Muscle in Pompe Disease
-
Salmeterol, a Beta-Agonist, Enhances Therapeutic Efficacy in Pompe Gene Therapy
-
Science and the law. Working through the patent problem.
-
Selective modification of antigen-specific T cells by RNA electroporation.
-
Sequences within the coding regions of clotting factor VIII and CFTR block transcriptional elongation.
-
Serial real-time luminescence imaging profiles differential kinetics and biodistibution of transgene expression from AAV serotype 1-5 vectors following adult and neonatal gene transfer
-
Shear degradation as a probe of microalgal exopolymer structure and rheological properties
-
Short-term cell/substrate contact dynamics of subconfluent endothelial cells following exposure to laminar flow.
-
Sialic Acids and Erythrocyte Interactions Are Critical Determinants of Cardiopulmonary Tropism Displayed by AAV Serotype 4
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Signal-transduction cascades as targets for therapeutic intervention by natural products.
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Single Vector AAV Approach to Genome Editing in Pompe Disease
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Single- and dual-fractal analysis of hybridization binding kinetics: biosensor applications.
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Smooth Muscle Cell Targeted RNA Aptamers for the Treatment of Vascular Disease
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Smooth Muscle Cell-targeted RNA Aptamer Inhibits Neointimal Formation.
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Solubilization of tricalcium phosphate by P(3HB) accumulating Azotobacter chroococcum MAL-201.
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Split luciferase complementation assay for the analysis of G protein-coupled receptor ligand response in Saccharomyces cerevisiae.
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Spontaneous bacterial cell lysis and biofilm formation in the colon of the Cape Dune mole-rat and the laboratory rabbit.
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Stabilization of D-amino acid oxidase and catalase in permeabilized Rhodotorula gracilis cells and its application for the preparation of alpha-ketoacids*.
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Statistical determination of steady state condition in bioremediation tests
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Steady ongoing hematological and immunological reconstitution achieved in ADA-deficiency patients treated by stem cell gene therapy with no myelopreparative conditioning
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Stimulus responsive elastin biopolymers: Applications in medicine and biotechnology.
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Streptavidin-biotin binding energetics.
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Structural Mapping of AAV9 Antigenic Sites and the Engineering of Immune Escape Variants
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Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice
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Structure-Guided Iterative Evolution of Antigenically Advanced AAV Variants for Therapeutic Gene Transfer
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Studies on the preparation of chitosan microcarriers cross-linked by oxidized lactose and culture of primary hepatocytes.
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Study of substrate specificity of RNR-exoribonucelases using hybrid proteins
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Subgenomic Flaviviral RNA Elements Enhance rAAV Vector mRNA Stability and Transduction
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Substrate (gelatin) gel electrophoretic method for analysis of protease activity in insect (Sf-9) cells
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Successful AAV8 readministration: Suppression of capsid-specific neutralizing antibodies by a combination treatment of bortezomib and CD20 mAb in a mouse model of Pompe disease.
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Successful immunization with a single injection of non-integrating lentiviral vector.
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Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice.
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Synchronous luminescence: A simple technique for the analysis of hydrolysis activity of the fragile histidine triad protein
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Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease.
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Synergistic and Tunable Gene Activation by Combinations of Synthetic Transcription Factors
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Synergistic effect of shear stress and streptavidin-biotin on the expression of endothelial vasodilator and cytoskeleton genes.
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Synthetic Biology: Caught Between Property Rights, the Public Domain, and the Commons
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Synthetic Biology: Reports from CSHA 2016 and More.
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Systemic Correction of Murine Glycogen Storage Disease Type IV by an AAV-Mediated Gene Therapy.
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Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.
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Systemic Delivery of AAVB1-GAA Gene Therapy for Respiratory Pathology in Pompe Disease
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Systemic Expression of Translatable Circular Rnas Using Recombinant AAV Vectors
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Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector.
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Systemic production of human granulocyte colony-stimulating factor in nonhuman primates by transplantation of genetically modified myoblasts.
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Targeted In Vivo Manipulation of Murine T-Cells Using a Newly Evolved AAV Capsid Mutant
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Targeted gene therapy for rat glomerulonephritis using HVJ-immunoliposomes.
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Targeting Muscle Satellite Cells for In Vivo Gene Editing With Adeno-Associated Viral Vectors for Duchenne Muscular Dystrophy
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Technology developments in endovascular treatment of intracranial aneurysms.
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Technology evaluation: BLP-25, Biomira Inc.
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Technology evaluation: CEA-TRICOM, Therion Biologics Corp.
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Technology evaluation: ISIS-2503, Isis Pharmaceuticals.
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Technology evaluation: Rexin-G, Epeius Biotechnologies.
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Technology evaluation: Stem-cell therapy, Aastrom Biosciences Inc.
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Technology evaluation: Theratope, Biomira Inc.
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Technology evaluation: VEGF165 gene therapy, Valentis Inc.
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Technology evaluation: gene therapy (IL-2), Valentis Inc.
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Technology evaluation: ipilimumab, Medarex/Bristol-Myers Squibb.
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Th1 cytokine interferon gamma improves response in HER2 breast cancer by modulating the ubiquitin proteasomal pathway.
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The AAV vector toolkit: poised at the clinical crossroads.
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The ELF3 transcription factor is associated with an epithelial phenotype and represses epithelial-mesenchymal transition
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The Human Silencing Hub (HUSH Complex) Is a Potent Regulator of AAV Transgene Silencing
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The Image Gently ALARA CT summit on new CT technologies for children.
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The Membrane-Associated Accessory Protein (MAAP) is Essential for Rapid Extracellular Secretion of Adeno-Associated Viruses
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The New Epigenome-Editing Approach for Targeting Dysregulated SNCA Expression: Novel Target Validation for Next-Generation Drug Discovery
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The New Frontier in the Development of LOAD Therapies Targeting APOE
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The Path to Progress Preclinical Studies of Age-Related Neurodegenerative Diseases: A Perspective on Rodent and hiPSC-Derived Models.
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The Removal of Volatile Ketone Mixtures from Air in Biofilters
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The U.S. Culture Collection Network Responding to the Requirements of the Nagoya Protocol on Access and Benefit Sharing
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The development of new biotechnologies using metalloprotein design.
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The development of optical nanosensors for biological measurements.
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The early years of single photon emission computed tomography (SPECT): an anthology of selected reminiscences.
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The effect of an applied potential on the activity of carbonic anhydrase immobilized on graphite rods.
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The effect of osmotic stress on the production of nukacin ISK-1 from Staphylococcus warneri ISK-1.
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The effect of packing hydrophilization on bacterial attachment and the relationship with the performance of biotrickling filters.
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The impact of genomics on the biotechnology industry.
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The influence of DNA sequence on the immunostimulatory properties of plasmid DNA vectors.
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The quantity and quality of worldwide new drug introductions, 1982-2003.
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The rational design of allosteric interactions in a monomeric protein and its applications to the construction of biosensors.
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The removal of volatile ketone mixtures from air in biofilters
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The role of single-cell analyses in understanding cell lineage commitment.
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The synthetic utility of KDPGal aldolase
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The transcapsidation of AAV serotypes
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Therapeutic Benefit of Autophagy Modulation in Pompe Disease.
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Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.
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Time-Dependent Changes in ON Bipolar Cell Transcriptomes before and after Genetic Rescue from Rod Degeneration
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Tissue Engineered Human Skeletal Muscle as a Pre-Clinical Model for AAV Treatment of Pompe Disease
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Tissue engineering of functional skeletal muscle: challenges and recent advances.
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Toluene degradation in the recycle liquid of biotrickling filters for air pollution control.
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Total RNA isolation by a rapid centrifugation method.
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Toward predictive engineering of gene circuits.
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Training physicians for careers in clinical research: a tailored educational experience.
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Transcription factor decoy for NFkappaB inhibits TNF-alpha-induced cytokine and adhesion molecule expression in vivo.
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Transcriptional regulation improves the throughput of three-hybrid counter selections in Saccharomyces cerevisiae.
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Transcriptional regulation of changes in growth, cell cycle, and gene expression of Saccharomyces cerevisiae due to changes in buoyancy.
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Transduction of human antigen-presenting cells with integrase-defective lentiviral vector enables functional expansion of primed antigen-specific CD8(+) T cells.
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Transfection of RNA encoding tumor antigens following maturation of dendritic cells leads to prolonged presentation of antigen and the generation of high-affinity tumor-reactive cytotoxic T lymphocytes.
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Transgene Immunogenicity Has Significant Effects on Gene Therapy in a Mouse Model of Adult Polyglucosan Body Disease
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Transient-state behavior of a biofilter removing mixtures of vapors of MEK and MIBK from air.
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Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.
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Transmembrane transport of marker molecules and cell membrane kinetics during electropermeabilization
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Treatment of Sepsis by Neutralization of Extracellular Histones with Nucleic Acid Aptamers
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Tumor cells cotransduced with B7.1 and gamma-IFN induce effective rejection of established parental tumor.
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Turning lemons into lemonade: learning from negative studies in cancer immunotherapy.
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U.S. Department of Health and Human Services National Institutes of Health Recombinant DNA Advisory Committee: Minutes of meeting: December 6, 2001
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Ultra-high expression of a thermally responsive recombinant fusion protein in E. coli.
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Uncertain benefit: investigators' views and communications in early phase gene transfer trials.
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Understanding metabolism with flux analysis: From theory to application.
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Underwater life support based on immobilized oxygen carriers.
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Universal purification of AAV serotypes 1-5 modified to contain a heparin binding epitope
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Unlocking Avian AAV Transduction in Mammalian Cells and Tissues for Immune Evasion and Redosing
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Use of Sepharose 4B for preparative scale fractionation of eukaryotic messenger RNA's.
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Using Functional SNPs to Calculate a Polygenic Risk Score for LOAD
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VB4-845, a conjugated recombinant antibody and immunotoxin for head and neck cancer and bladder cancer.
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VSMC Specific Aptamer Ligands for the Next Generation of DES to Prevent Neointimal Formation
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Validation of therapeutic strategies in a novel compound heterozygote model of Methylmalonic Acidemia
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Vector Genome Length and Self-Complementarity Affect AAV Capsid Uncoating
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Vector-related tumorigenesis not found in ornithine transcarbamylase-deficient mice.
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Viral vector gene delivery of the novel chaperone protein SRCP1 to modify insoluble protein in in vitro and in vivo models of ALS.
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Viruses for the treatment of malignant glioma.
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When is an adjuvant not an adjuvant?
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When is an adjuvant not an adjuvant? Overview.
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Whole-body correction of Pompe disease by AAV liver-mediated gene transfer of engineered secretable GAA transgenes
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Wide Spread Adenoviral Vector Delivery to a Cardiac Allograft Utilizing an Ex Vivo Perfusion Storage Strategy
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Wolfgang Barz [formula omitted] CoĊstitutive and Elicitation Induced Metabolism of Isoflavones and Pterocarpans in Chickpea (Cicer arietinum) Cell Suspension Cultures
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X-linked inhibitor of apoptosis protein-mediated attenuation of apoptosis, using a novel cardiac-enhanced adeno-associated viral vector.
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X-ray crystallographic studies of streptavidin mutants binding to biotin.
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ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering.
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Zero incidence of adeno-associated virus serotype 9 (AAV9) antibodies in a cohort of spinal muscular atrophy (SMA) type 1 patients screened in STR1VE, a pivotal phase 3 study of AVXS-101
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β-Cyclodextrin-containing polymer treatment of cutaneous lupus and influenza improves outcomes.
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Keywords of People
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Huang, Linfeng,
Associate Professor of Biology at Duke Kunshan University,
DKU Faculty
-
Kelsoe, Garnett H.,
James B. Duke Distinguished Professor of Immunology,
Integrative Immunobiology
-
Reed, Shelby Derene,
Professor in Population Health Sciences,
Duke Science & Society
-
Ridley, David Blaine,
Professor of the Practice of Business Administration,
Duke Science & Society
-
Sexton, Steven E.,
Mark and Lynne Florian Associate Professor of Public Policy,
Economics