Publications

• The 2024 Nobel Prize: Impact of the Discovery of miRNA on the Field of Gene Therapy

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

• Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases

• AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys

• Kenneth I. Berns, MD, PhD [1938-2024]

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

• Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders

• Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency

• Alpha-1 Antitrypsin Deficiency

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

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

• Gene therapy for alpha-1 antitrypsin deficiency: an update

• Nicholas Muzyczka, PhD [1947-2023]

• Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop

• Nicholas Muzyczka, PhD

• Defining Scholarship for Today and Tomorrow

• Secretion of functional α1-antitrypsin is cell type dependent: Implications for intramuscular delivery for gene therapy

• Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice

• Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

• AAV gene therapy for Tay-Sachs disease

• Gene Therapy for Rare Neurological Disorders

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

• ESGCT 2021: Virtually Pan-European

• In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis

• Base Editing to the Rescue

• Supporting Families Considering Participation in a Clinical Trial: Parent-Provider Perspectives

• Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

• Large-scale molecular epidemiological analysis of AAV in a cancer patient population

• Common pathways to Dean of Medicine at U.S. medical schools

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

• In Reply to Ramotshwana et al

• Liver targeting with rAAV7: balancing tropism with immune profiles

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

• Engraftment of Human Hepatocytes in the PiZ-NSG Mouse Model

• Accelerated Graduation and the Deployment of New Physicians During the COVID-19 Pandemic

• Volume and Infusion Rate Dynamics of Intraparenchymal Central Nervous System Infusion in a Large Animal Model

• Impact on Women's Health: Gene Therapy in Gynecologic Oncology

• Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

• A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna

• Women's Representation Among Members and Leaders of National Medical Specialty Societies

• 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

• New Horizons for Immune Gene Therapy

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

• The Year in Review: The Top Five Papers of 2018

• What the Gene Therapy Community Should Do About Sexual Harassment

• Assuring Integrity in the Residency Match Process

• Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

• Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy

• A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates