Terence Flotte, MD

• Lung endothelial transduction in a patient that succumbed to acute respiratory distress syndrome following high-dose rAAV9 gene therapy

• A single amino acid variant in the variable region I of AAV capsid confers liver detargeting

• Gene therapy then and now: A look back at changes in the field over the past 25 years

• 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

• The Road Less Traveled: Slow but Steady Progress Toward Cystic Fibrosis Gene Therapy by the UK Respiratory Gene Therapy Consortium

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

• Intrathecal gene therapy for neurologic disease in humans

• 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

• Lived Experience with Gene Therapy

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

• Annual Editor's Letter

• Special Editorial Feature: Learning from Our Failures

• 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