Molecular Therapy

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  Publication Venue For

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