Human Gene Therapy

Title Publication Date Language Citations
Prevalent and Disseminated Recombinant and Wild-Type Adeno-Associated Virus Integration in Macaques and Humans2023/11/06English
Kenneth I. Berns, MD, PhD [1938–2024]2024/03/01English
Akouos Therapy Restores 11-Year-Old Boy's Hearing After 1 Month2024/03/01English
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases2024/04/08English
Correction to: Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis, by Han et al. Hum Gene Ther 2024;35(3-4):93–103; doi: 10.1089/hum.2023.208.2024/03/25English
A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies2024/04/09English
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop2023/02/01English
Nicholas Muzyczka, PhD [1947–2023]2023/02/01English
Secretion of 4-1BB Ligand Crosslinked to PD-1 Checkpoint Inhibitor Potentiates Chimeric Antigen Receptor T Cell Solid Tumor Efficacy2023/04/20English
ESGCT Abstract Author Index2024/02/01English
ESGCT 30th Annual Congress In collaboration with SFTCG and NVGCT Brussels, Belgium October 24–27, 2023 Abstracts2024/02/01English
Acknowledgment of Reviewers 20232024/01/01English
Effects of Urocortin 2 Gene Transfer on Glucose Disposal in Insulin-Resistant db/db Mice on Metformin2023/08/01English
Direct Comparison of Epifluorescence and Immunostaining for Assessing Viral Mediated Gene Expression in the Primate Brain2023/03/01English
Acknowledgment of Reviewers 20222023/01/01English
Interview with Dr. Florian Eichler2023/01/01English
Fyodor Urnov Articulates New Model for Developing, Financing Therapies2023/01/01English
Orchard Therapeutics Gains First U.S. Approval for a Metachromatic Leukodystrophy Gene Therapy2024/04/01English
Remembrances of Kenneth Berns, PhD2024/04/01English
Corrigendum2000/10/01English
National Institutes of Health Recombinant DNA Advisory Committee (RAC) Building 45/Natcher Conference Center, Conference Room D Bethesda, Maryland June 14-15, 20012001/08/10English
Abstract Author Index1999/03/20English
Correction2002/02/10English
Future Meetings of the NIH Recombinant DNA Advisory Committee (RAC) and Gene Therapy Policy Conferences (GTPC)1999/08/10English
Regulatory Issues: Future Meetings of the NIH Recombinant DNA Advisory Committee (RAC) and Gene Therapy Policy Conferences (GTPC)2000/07/01English
Future Meeting Dates and Protocol Submission Deadlines for Public Recombinant DNA Advisory Committee (RAC) Review2002/01/20English
Knockout and Replacement Gene Surgery to Treat Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa2024/03/01English
Future Meeting Dates and Protocol Submission Deadlines for Public Recombinant DNA Advisory Committee (RAC) Review2001/03/20English
Regulatory Issues: Future Meeting Dates and Protocol Submission Deadlines for Public Recombinant DNA Advisory Committee (RAC) Review2000/10/01English
Future Meeting Dates and Protocol Submission Deadlines for Public Recombinant DNA Advisory Committee (RAC) Review2001/09/20English