Publications

• AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial

• Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial

• AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial

• Efficient Cas9 nuclease-based editing in skeletal muscle via lipid nanoparticle delivery

• The curious case of AAV immunology

• Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy

• Immune responses to central nervous system directed adeno-associated virus gene therapy: Does direct CNS delivery make a difference?

• Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques

• Genetic ablation of Sarm1 attenuates expression and mislocalization of phosphorylated TDP-43 after mouse repetitive traumatic brain injury

• Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. Reply

• Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy

• Regulatory T Cell Therapeutics for Neuroinflammatory Disorders

• Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer

• Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System

• Evaluating Readministration of Adeno-Associated Virus for Gene Therapy

• CAR-T Regulatory (CAR-Treg) Cells: Engineering and Applications

• Rational Use of Immunosuppressive Corticosteroids in Liver-Directed Adeno-Associated Virus Gene Therapy Studies

• AAV gene therapy for Tay-Sachs disease

• Immune Responses to Adeno-Associated Virus-Mediated CRISPR Therapy

• Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells

• Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction

• CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing

• Two-Plasmid Packaging System for Recombinant Adeno-Associated Virus

• Intralingual and Intrapleural AAV Gene Therapy Prolongs Survival in a SOD1 ALS Mouse Model

• Pollen-derived RNAs Are Found in the Human Circulation

• Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

• AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency

• Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study

• Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease

• Airway smooth muscle dysfunction in Pompe (Gaa-/- ) mice

• CAR T-Cell Therapy: Progress and Prospects

• Restrictive Lung Disease in the Cu/Zn Superoxide-Dismutase 1 G93A Amyotrophic Lateral Sclerosis Mouse Model

• Cellular Analysis of Silencing the Huntington's Disease Gene Using AAV9 Mediated Delivery of Artificial Micro RNA into the Striatum of Q140/Q140 Mice

• Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector

• Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan

• Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndrome

• Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy

• Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responses

• Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17e