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Publication Venue For
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Immunity to Cas9 as an Obstacle to Persistent Genome Editing..
28:1389-1391.
2020
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AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice..
27:1568-1585.
2019
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RNA-Modified T Cells Mediate Effective Delivery of Immunomodulatory Cytokines to Brain Tumors..
27:837-849.
2019
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It's All in the Delivery: Designing Hydrogels for Cell and Non-viral Gene Therapies..
26:2087-2106.
2018
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Boosting, Not Breaking: CRISPR Activators Treat Disease Models..
26:334-336.
2018
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Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice.
25:2150-2162.
2017
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Genome-editing Technologies for Gene and Cell Therapy..
24:430-446.
2016
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Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases.
23:523-532.
2015
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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).
21:2130.
2013
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Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients..
21:1718-1726.
2013
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Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases..
21:266-268.
2013
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Reading frame correction by targeted genome editing restores dystrophin expression in cells from duchenne muscular dystrophy patients.
21:1718-1726.
2013
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Notable reduction in illegitimate integration mediated by a PPT-deleted, nonintegrating lentiviral vector.
19:547-556.
2011
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Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: A strategy for broad clinical application.
18:1907-1916.
2010
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Successful immunization with a single injection of non-integrating lentiviral vector..
15:1716-1723.
2007
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Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II..
14:822-830.
2006
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Mesenchymal stem cells overexpressing Akt dramatically repair infarcted myocardium and improve cardiac function despite infrequent cellular fusion or differentiation..
14:840-850.
2006
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Multiple innate inflammatory responses induced after systemic adenovirus vector delivery depend on a functional complement system..
14:588-598.
2006
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A novel antidote-controlled anticoagulant reduces thrombin generation and inflammation and improves cardiac function in cardiopulmonary bypass surgery..
14:408-415.
2006
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Adeno-associated virus serotypes: vector toolkit for human gene therapy..
14:316-327.
2006
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Polyclonal long-term MFGS-gp91phox marking in rhesus macaques after nonmyeloablative transplantation with transduced autologous peripheral blood progenitor cells..
14:202-211.
2006
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Vector-related tumorigenesis not found in ornithine transcarbamylase-deficient mice..
14:1-2.
2006
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219. Persistent Correction of Hyperphenylalaninemia Following Liver-Directed, rAAV2/8-Mediated Gene Therapy for Murine Phenylketonuria (PKU).
13:S84-S84.
2006
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427. Adenovirus Mediated GRP94/gp96 Expression in Treatment of Neuroblastoma.
13:S164-S164.
2006
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954. High Intensity Focused Ultrasound (HIFU) Induced Trans-Gene Activation in Solid Tumors.
13:S368-S368.
2006
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Fully deleted adenovirus persistently expressing GAA accomplishes long-term skeletal muscle glycogen correction in tolerant and nontolerant GSD-II mice..
13:127-134.
2006
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Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II..
12:876-884.
2005
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HSV oncolytic therapy upregulates interferon-inducible chemokines and recruits immune effector cells in ovarian cancer..
12:789-802.
2005
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Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter..
11:889-898.
2005
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Retargeting mobile group II introns to repair mutant genes..
11:687-694.
2005
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Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II..
11:57-65.
2005
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Methotrexate and cytarabine inhibit progression of human lymphoma in NOD/SCID mice carrying a mutant dihydrofolate reductase and cytidine deaminase fusion gene..
10:574-584.
2004
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Ribozyme-mediated induction of apoptosis in human cancer cells by targeted repair of mutant p53 RNA..
10:365-372.
2004
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Uncertain benefit: investigators' views and communications in early phase gene transfer trials..
10:225-231.
2004
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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..
9:757-764.
2004
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A quantitative method for measuring gene expression magnitude and volume delivered by gene therapy vectors..
9:625-631.
2004
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HIV-1 Vif: counteracting innate antiretroviral defenses..
8:525-527.
2003
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GENIS: gene expression of sodium iodide symporter for noninvasive imaging of gene therapy vectors and quantification of gene expression in vivo..
8:508-518.
2003
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Catheter-based intracoronary myocardial adenoviral gene delivery: importance of intraluminal seal and infusion flow rate..
8:306-313.
2003
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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..
7:467-477.
2003
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Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector..
7:193-201.
2003
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Optimizing aptamer activity for gene therapy applications using expression cassette SELEX..
6:30-34.
2002
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Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model..
5:780-787.
2002
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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..
5:436-446.
2002
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Generation of species cross-reactive aptamers using "toggle" SELEX..
4:567-573.
2001
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Generation of recombinant adeno-associated virus vectors by a complete adenovirus-mediated approach..
3:787-792.
2001
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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..
3:54-60.
2001
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Combination treatment of murine tumors by adenovirus-mediated local B7/IL12 immunotherapy and radiotherapy..
2:195-203.
2000
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Enhancing RNA repair efficiency by combining trans-splicing ribozymes that recognize different accessible sites on a target RNA..
2:245-255.
2000
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Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors..
2:16-25.
2000
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A Human iPSC-Derived Neuron and Cardiomyocyte Platform for Assessing Novel Recombinant Adeno-Associated Viral (AAV) Vectors
2022
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A Newly Evolved AAV Variant Enables Potent Gene Transfer in Kidneys of Multiple Species
2022
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A Novel Human Liver Chimeric Mouse Model Lacking the Murine AAVR Gene for Validation of AAV Gene Therapy Vectors
2022
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AAV Capsid Dynamics at the 5-Fold Pore Controls Genome Release
2022
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AAV Gene Therapy Effectively Transduces the Central Nervous System and Prevents Polyglucosan Body Formation in Adult Polyglucosan Body Disease Mice
2022
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AAV Hijacks Cellular Secretion Machinery for Efficient Viral Egress
2022
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APOE-Targeted Epigenome Therapy for Alzheimer's Disease
2022
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Bimodular CRISPR/Cas-lentiviral Vector for Modeling Diseases
2022
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Efficacy of Genome Editing in Infant Mice with Glycogen Storage Disease Type Ia
2022
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Enhanced CRISPR/Cas9 Genome Editing in Heart and Skeletal Muscle with a Potent New AAV Variant
2022
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Evolution of a New AAV Variant with Murine T Lymphocyte Tropism Using the MHC-Ib Molecule H2-Q7 as a Receptor
2022
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Furin is a Host Factor Restricting Adeno-Associated Virus 4 Transduction
2022
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Host Transcription Factors and Co-Opted Signaling Pathways Orchestrate Epigenetic Modulation of the AAV Vector Genome
2022
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Liver Directed AAV Gene Therapy Reverses Progression of Glycogen Storage Disease Type IX gamma 2 in Mice
2022
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Long-Term Correction of Glycogen Storage Disease Type III by AAV-Mediated Gene Therapy Using a Dual Promoter to Express Bacterial Pullulanase
2022
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Novel Gene Supplementation, Genome Editing and Cellular Therapeutic Approaches to Treat Glutaric Aciduria Type I
2022
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Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Initial 104-Week Experience
2022
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Pre-Existing Humoral Immunity to AAV Capsids: Species and Age-Related Differences in Anti-Capsid ELISA and Neutralizing Antibody Assays
2022
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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
2022
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STRV5, a Liver Detargeted AAV Capsid Demonstrates Improved Safety Profile After Intravenous Administration of High Doses in Non-Human Primates
2022
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Targeted In Vivo Manipulation of Murine T-Cells Using a Newly Evolved AAV Capsid Mutant
2022
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Unlocking Avian AAV Transduction in Mammalian Cells and Tissues for Immune Evasion and Redosing
2022
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A CRISPR/Cas9 Genome Wide Screen and Cryo-EM Structure Reveal Receptor Switching by a Synthetic AAV Variant
2021
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A CRISPRa Screen Identifies Transcription Factors That Can Silence or Activate rAAV Genomes
2021
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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
2021
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CRISPR/Cas9 Inducible Systems for Neurodegenerative Disease
2021
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Characterization of Liver Pathology in a Novel GSD IX y2 Mouse Model
2021
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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
2021
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Development of AAV-Based CRISPR/Cas9 Therapies for Correcting Duchenne Muscular Dystrophy by Targeted Genomic Integration
2021
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Distinct Functional Roles for AAV Capsid Surface Epitopes Revealed through Structure-Guided Evolution
2021
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Downregulation of SNCA Expression as a Therapoetic Approach for Parkinson's Disease and Other Synucleinopathies: New Target Validation for Next-Generation Drug Discovery
2021
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Epigenome-Base Strategies for Efficient and Safe Editing of APOE epsilon 4 Allele: Implications for Alzheimer's Disease
2021
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Evolving Synthetic AAV Variants for Genome Editing in Immune Cell Populations
2021
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Mechanistic Elucidation of Adenovirus Mediated Enhancement of Recombinant Adeno-Associated Virus (rAAV) towards Efficient Low Dose Gene Therapy
2021
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Policies and Pitfalls: What the Alzheimer's Community Can Learn from the Deployment of Disease-Modifying Therapies for Multiple Sclerosis Patients
2021
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Single Vector AAV Approach to Genome Editing in Pompe Disease
2021
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Subgenomic Flaviviral RNA Elements Enhance rAAV Vector mRNA Stability and Transduction
2021
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The Human Silencing Hub (HUSH Complex) Is a Potent Regulator of AAV Transgene Silencing
2021
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The New Frontier in the Development of LOAD Therapies Targeting APOE
2021
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Time-Dependent Changes in ON Bipolar Cell Transcriptomes before and after Genetic Rescue from Rod Degeneration
2021
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Using Functional SNPs to Calculate a Polygenic Risk Score for LOAD
2021
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A Combination Immunosuppression Treatment with Bortezomib and an Anti-CD20 mAb Enables Successful Re-Administration of AAV8 Vector in Mice
2020
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A Novel Approach for Rapid and Transient Depletion of Pre-Existing Neutralizing Antibodies Against AAV Vectors
2020
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A Novel Capsid for Smooth Muscle Pathology in Pompe Disease
2020
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AAV-Mediated Gene Therapy for Glycogen Storage Disease Type III Using a Bacterial Glycogen Debranching Enzyme
2020
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Allele Specific Knockdown of APOE e4 Expression: A Novel Platform for Precision Gene Therapy in Alzheimer's Disease
2020
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An Isogenic hiPSC-Derived Model System for Establishing Gene Therapy Approaches in Alzheimer's Disease
2020
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CRISPR-Mediated Gene Correction in a Severe Humanized Mouse Model of Duchenne Muscular Dystrophy
2020
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Cross-Species Evolution of Synthetic AAV Strains for Clinical Translation
2020
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Development of Lentiviral Platform for Allele-Specific Targeted Down-Regulation of APOEe4 Expression: Epigenetic Therapy for Precision Medicine in Alzheimer's Disease
2020
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Engineering Backsplicing Introns for Robust Expression of Synthetic Circular RNAs from AAV Vectors
2020
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Epigenetic Downregulation of ApoE4 for Alzheimer's Disease
2020
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Evolving New AAV Strains that Demonstrate AAVR Independence
2020
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Implications of Gene Therapy for Alzheimer's
2020
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Inhibition of Caspase-3 Improves Electrotransfer Efficiency and Cell Viability
2020
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Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Analyses of Safety and Secondary Endpoints
2020
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Targeting Muscle Satellite Cells for In Vivo Gene Editing With Adeno-Associated Viral Vectors for Duchenne Muscular Dystrophy
2020
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The Membrane-Associated Accessory Protein (MAAP) is Essential for Rapid Extracellular Secretion of Adeno-Associated Viruses
2020
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Transgene Immunogenicity Has Significant Effects on Gene Therapy in a Mouse Model of Adult Polyglucosan Body Disease
2020
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A CRISPR Screen Identifies Apical Polarity Determinant Crumbs 3 as an AAV Host Restriction Factor
2019
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AAV Gene Therapy in a Canine Model of MPS1 Prevents and Reverses Corneal Blindness
2019
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AAV-Mediated Deletion of a Large Mutational Hotspot for Treatment of Duchenne Muscular Dystrophy
2019
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Correction of Glycogen Storage Disease Type III with an AAV Vector Encoding a Bacterial Glycogen Debranching Enzyme
2019
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Design Principles for AAV Mediated Circular RNA Expression in the Brain
2019
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Engineering A Humanized AAV8 Capsid through Iterative Structure-Guided Evolution
2019
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Exploring Novel Aptamer-Heparin Combinations to Improve Anticoagulation Therapy in Cardiopulmonary Bypass
2019
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Inhalation of an RNA Aptamer Targeting Extracellular Histones Protects from Acute Lung Injury
2019
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Integrase-Deficient Lentiviral Vector for Efficient CRISPR/Cas9-Mediated Gene and Epigenome-Editing Applications
2019
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Long-Term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy
2019
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Mass Spectrometric Identification of Host Factors that Restrict AAV Vector Genome Transcription
2019
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Physical Positioning Dramatically Improves Brain Transduction after Intrathecal Infusion of AAV9
2019
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Reporting Long Term Survival Following Precision Tumor-Targeted Gene Delivery to Advanced Chemotherapy-Resistant Malignancies: An Academic Milestone
2019
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Respiratory Directed Gene Therapy to Silence SOD1 Prolongs Survival in the SOD1 ALS Mouse
2019
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Salmeterol with Liver Depot Gene Therapy Reversed Biochemical and Autophagic Abnormalities of Skeletal Muscle in Pompe Disease
2019
-
The New Epigenome-Editing Approach for Targeting Dysregulated SNCA Expression: Novel Target Validation for Next-Generation Drug Discovery
2019
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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
2018
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Bezafibrate Enhances AAV Gene Therapy in Glycogen Storage Disease Type Ia
2018
-
Capsid-Glycan Receptor Interactions Influence AAV Transport across the Blood-Brain Barrier
2018
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Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. Aureus Cas9
2018
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Expression of CRISPR-Cas9 Single Guide RNA's Using Small tRNA Promoters
2018
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Long-Term Evaluation of Genome Editing Outcomes for Duchenne Muscular Dystrophy
2018
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Mapping and Engineering Functional Domains of the Assembly Activating Protein of Adeno-Associated Viruses
2018
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RNF121 is a Key Transcriptional Regulator of AAV Genome Expression
2018
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Systemic Expression of Translatable Circular Rnas Using Recombinant AAV Vectors
2018
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VSMC Specific Aptamer Ligands for the Next Generation of DES to Prevent Neointimal Formation
2018
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Wide Spread Adenoviral Vector Delivery to a Cardiac Allograft Utilizing an Ex Vivo Perfusion Storage Strategy
2018
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Inhibition of Extracellular Histones in Sepsis-Induced Cardiovascular Function
2018
-
Rna Inhibitors of Nuclear Proteins Implicated in Multiple Organ Dysfunction Syndrome
2018
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Discovery of a Neurotropic Footprint That Enables AAV Transport Across the Blood-Brain Barrier
2017
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Disruption of Water Homeostasis Affects AAV Transport Across the Blood-Brain Barrier
2017
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Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. aureus Cas9
2017
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Editing out Five SERPINA1 Paralogs to Create a New Mouse Model of Genetic Emphysema
2017
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Effect of AAV Gene Therapy on Hepatic Tumor Induced by Deficiency of Glucose-6-Phosphatase
2017
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Expanding the CNS Transduction Profile of AAV4 by Structure-Guided Evolution
2017
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Liver-Mediated Gene Therapy with an Engineered Secretable GAA Transgene Results in Whole-Body Correction of Pompe Disease
2017
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Long-Term Characterization of In Vivo Genome Editing in a Mouse Model of Duchenne Muscular Dystrophy
2017
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Salmeterol, a Beta-Agonist, Enhances Therapeutic Efficacy in Pompe Gene Therapy
2017
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Structural Mapping of AAV9 Antigenic Sites and the Engineering of Immune Escape Variants
2017
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Structure-Guided Iterative Evolution of Antigenically Advanced AAV Variants for Therapeutic Gene Transfer
2017
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Systemic Delivery of AAVB1-GAA Gene Therapy for Respiratory Pathology in Pompe Disease
2017
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Treatment of Sepsis by Neutralization of Extracellular Histones with Nucleic Acid Aptamers
2017
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167. Genome Editing to Generate the First Mouse Model of Alpha-One Antitrypsin Deficiency, the Leading Cause of Genetic COPD
2016
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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)
2016
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30. Modulation of Intracellular Calcium Enhances AAV Transduction in the CNS
2016
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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
2016
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381. Prohypertrophic Agents Enhance the Response to Gene Therapy in Pompe Disease
2016
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482. Local and Systemic Gene Editing in a Mouse Model of Duchenne Muscular Dystrophy
2016
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49. Minimum Effective Dose of Liver-Targeted Gene Therapy for Pompe Disease Reduced 10-Fold from Prior Estimates
2016
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504. Restoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular Dystrophy
2016
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727. Characterizing the Adeno-Associated Virus 1 Sialic Receptor Binding Site and Its Overlap with Antigenic Epitopes
2016
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8. Further Characterization of U2 snRNP Mediated Restriction of AAV Vector Transduction
2016
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93. Development of AAVidua Vector for the Treatment of Cornea Associated Vision Loss in Bone Marrow Treated-Hurler Patients
2016
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Aquaporins and CSF Flux Are Critical Determinants of AAV Mediated CNS Gene Transfer
2016
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14. Next Generation AAV Vectors for Limiting Systemic Leakage and Improving Safety Following CNS Administration
2015
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265. Polysialic Acid as a Novel Regulator of AAV Tropism in the Developing Brain
2015
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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
2015
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302. Combinatorial Engineering of a Receptor Footprint on AAV Serotype 4 Yields Novel Vectors
2015
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38. Pharmacological Regulation of Vesicular Trafficking as a Strategy to Enhance Recombinant AAV Transduction
2015
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397. Correction of the Dystrophin Gene By the CRISPR/Cas9 System in a Mouse Model of Muscular Dystrophy
2015
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41. U2 snRNP Spliceosome Proteins Block Recombinant AAV Vector Transduction
2015
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42. Differential Transduction Profiles of AAV Vectors in a Mouse Model of Human Glycosylation
2015
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472. Low-Shear Sodding of an Implantable Expanded Polytetrafluoroethylene Scaffold with a Rat Adipose-Derived Stromal-Vascular Fraction
2015
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485. Targeted Epigenome Editing by CRISPR/Cas9-Based Repressors for Silencing of Distal Regulatory Elements
2015
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59. Multiplex Gene Activation by CRISPR/Cas9-Based Transcription Factors for the Direct Conversion of Fibroblasts to a Neuronal Phenotype
2015
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61. Vascular Smooth Muscle Cell RNA Aptamers for the Treatment of Cardiovascular Disease
2015
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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)
2015
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701. Long-Term Complications of Glycogen Storage Disease Type IA in the Dog Model Treated With Gene Replacement Therapy
2015
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A Novel Aptamer Targeting Agent for Prostate Cancer
2015
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Lymphatic Transport Mediates Systemic Leakage of Peripherally Administered AAV9 Vectors
2015
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Bioengineered Skeletal Muscle With Functional Stem Cell Pool and Capacity for Vascular Integration and Maturation In Vivo
2014
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CRISPR/Cas9-Based Transcriptional Repressors for Control of Human Gene Expression
2014
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Cell-Targeted RNA-Based Therapies for Cardiovascular Disease
2014
-
Characterizing Essential Receptor Binding Residues for AAV1
2014
-
Gene Therapy for Heart Disease Using Electrically Active Fibroblasts
2014
-
Human Pluripotent Stem Cell-Derived Cardiac Tissue Patch for Use in Cell-Based Cardiac Therapy
2014
-
Mapping the Antigenic Structure of Adeno-Associated Virus Serotypes 8 and 9
2014
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Mutation Detection Following Non-Homologous End Joining (NHEJ): A Comparison of Different Semi Quantitative and Quantitative Methods
2014
-
Neonatal rAAV2/8 Vector Administration Does Not Prevent Therapeutic Response To Repeat Vector Injection in Adult Pah(enu2/enu2) Mice
2014
-
Nucleic-Acid Binding Polymers as Anti-Inflammatory Agents
2014
-
Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID)
2014
-
Regional-CNS MiniPromoters for AAV Are Identified in a High Through-Put Pipeline
2014
-
Tissue Engineered Human Skeletal Muscle as a Pre-Clinical Model for AAV Treatment of Pompe Disease
2014
-
Adjunctive beta 2-Agonist Therapy Enhances Trafficking of Acid alpha-Glucosidase to Lysosomes in the Skeletal Muscle of Mice with Pompe Disease
2013
-
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
2013
-
Integrins Play a Pivotal Role in Transvascular Transport and Tissue Uptake of AAV9 In Vivo
2013
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Pathogenesis of Growth Failure in Murine and Canine Glycogen Storage Disease-Ia and Partial Reversal of Growth Failure Using Adeno-Associated Viral Vector Therapy
2013
-
Re-Engineering Capsid-Glycan Interactions Expands the CNS Transduction Profile of AAV Serotype 4
2013
-
Sialic Acids and Erythrocyte Interactions Are Critical Determinants of Cardiopulmonary Tropism Displayed by AAV Serotype 4
2013
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Smooth Muscle Cell Targeted RNA Aptamers for the Treatment of Vascular Disease
2013
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Synergistic and Tunable Gene Activation by Combinations of Synthetic Transcription Factors
2013
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Altering Tissue Glycan Patterns Improves the Transduction Profile of AAV9 Vectors
2012
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Aptamer-siRNA Chimera Inhibitors of Intimal Hyperplasia
2012
-
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
2012
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Increased Frequency of Permanent Integration Following Liver-Directed Gene Therapy with rDNA-AAV2/8 In Murine Phenylketonuria
2012
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Vector Genome Length and Self-Complementarity Affect AAV Capsid Uncoating
2012
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Desensitization through Immunomodulatory Gene Therapy Suppresses the Antibody Response to Enzyme Replacement Therapy in Murine Pompe Disease
2009
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Imaging Trafficking Dynamics of Ultra-Pure Single Adeno-Associated Virus in Live Cells
2009
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Immune Reconstitution after Gene Therapy for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID)
2009
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Mechanism of Shortened Bones in Mucopolysaccharidosis VII
2009
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RNA Transfected Dendritic Cell Vaccines Targeting Human Cytomegalovirus Antigens in Patients with Glioblastoma
2009
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1028. Use of Antisense Oligonucleotides To Restore Activity in Mutant p53
2006
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104. Role of the HI Loop in the Adeno- Associated Virus (AAV) Life Cycle
2006
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1064. Haute Couture Vectors: Solving Clinical Problems with Custom Designed AAV Vectors
2006
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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
2006
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19. A New, Plasmid-Based Adenoviral Vectoring System Derived from the Highly Immunogenic, Human Ad4 Strain
2006
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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)
2006
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425. Translation of Enhanced Dendritic Cell Vaccines Using a CID-Inducible CD40 Receptor
2006
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487. Liver-Targeted Gene Therapy in Glycogen Storage Disease Type Ia (GSD-Ia) Requires Widespread, Regulated Glucose-6-phosphatase
2006
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563. Correction of Glycogen Storage Disease Type II by Systemic Delivery of an AAV2/8 Vector Containing a Muscle-Specific Promoter
2006
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586. Anti-HIV-1 Neutralizing Antibody Gene Delivery to Rhesus Macaques and Rodents: A Novel Vaccination Approach
2006
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7. Adeno-Associated Virus Type 2 (AAV2) Contains an Integrin alpha5beta1 Recognition Sequence Essential for Viral Cell Entry
2006
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739. Bioluminescent Virion Shells: A Novel Tool for Visualization of Viral Vectors in Animal Tissue
2006
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83. Cell_Type Specific Delivery of siRNAs with Aptamer_siRNA Chimeras
2006
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From crystal structure to clinic: highlights of the Tenth International Parvovirus Workshop.
2005
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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
2004
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A central role of RNA in developing therapeutic vaccines for cancer and infectious diseases
2004
-
Correction of glycogen storage disease type II (GSD II) with an adeno-associated virus 8 (AAV2/8) vector
2004
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Correction of glycogen storage disease type II by intramuscular administration of an adeno-associated virus 6 (AAV2/6) vector containing a muscle-specific promoter
2004
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Development of adeno-associated virus (AAV) vectors for gene therapy in the mouse model for glycogen storage disease type Ia (GSD-Ia)
2004
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Group I intron-mediated trans-splicing revision of mutant p53 RNA transcripts
2004
-
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
2004
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Pharmacological regulation of dendritic cell activation: Enhancing cancer vaccine efficacy
2004
-
Role of Vp2 in AAV packaging
2004
-
The transcapsidation of AAV serotypes
2004
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Universal purification of AAV serotypes 1-5 modified to contain a heparin binding epitope
2004
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5 ' exon replacement by spliceosome mediated RNA trans-splicing
2003
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A clinical attempt to treat JAK3-deficient SCID using retroviral-mediated gene transfer to bone marrow CD34+ cells
2003
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A genetically engineered plasmid encoding a zinc finger VEGF-activating transcription factor induces angiogenesis in the rabbits with hind-limb ischemia
2003
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Correction of glycogen storage disease type II (GSD II) by intramuscular administration of an adeno-associated virus (AAV) vector pseudotyped as AAV6
2003
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Corrective gene transfer into bone marrow CD34+cells for adenosine deaminase (ADA) deficiency: Results in four patients after one year of follow-up
2003
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Discontinuation of PEG-ADA replacement therapy in a patient with ADA-deficiency previously treated with retroviral-mediated, T cell-directed gene therapy
2003
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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
2003
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Transmembrane transport of marker molecules and cell membrane kinetics during electropermeabilization
2003