Terence Flotte, MD

• Analyzing clinical observations to better understand and manage immune responses to AAV gene therapies

• 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

• ESGCT 2022: The New Normal for the Gene Therapy Community

• Gene Therapy for Endocrine Disorders: A Promising Intervention

• 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

• Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient

• 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

• Precision of Exon Skipping with U7 Constructs

• ESGCT 2021: Virtually Pan-European

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

• Immune Responses to Recombinant Adenoviruses As Gene Therapy Vectors and COVID-19 Vaccines: A Two-Edged Sword

• Base Editing to the Rescue

• It Is Time to Call Anti-Asian Bias What It Is: Racism

• Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia

• Women Lead Gene Therapy Science in 2021

• 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

• CRISPR Keeps Things Fresh: Next-Generation Tools for Gene Editing

• In Reply to Ramotshwana et al

• Liver targeting with rAAV7: balancing tropism with immune profiles

• Gene and Cell Therapy for Inherited and Acquired Immune Deficiency

• Writing the Story of Gene Editing and CRISPR: An Interview with Kevin Davies, PhD

• Science Policy and Funding Implications of the 2020 U.S. Election

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

• Black Lives Matter to Gene Therapy

• SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

• Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy

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

• Barrie J. Carter: The Science and the Scientist

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

• Revisiting the "New" Inflammatory Toxicities of Adeno-Associated Virus Vectors

• A Message to Our Community in the Midst of the COVID-19 Pandemic

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

• Free Gene and Cell Therapy Services Offered by the National Heart, Lung, and Blood Institute of the National Institutes of Health

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

• 2020: Gene Therapy Enters Its Fourth Decade

• Gene Therapy Untangles the Problem of Chronic Traumatic Encephalopathy

• Ringing in the Changes