Table 1.

Resources Available for Gene Therapy Research

NIH resources

Gene Therapy Resource Program (GTRP) (www.gtrp.org)

National Heart, Lung, and Blood Institute (NHLBI)–supported program designed to facilitate translation of gene transfer applications into clinical interventions

Core facilities for preclinical and clinical good manufacturing practice–grade vectors (adeno-associated virus, adeno, and lentivirus), pharmacology, toxicology, and immunology studies, and a clinical coordinating center

Clinical trial performance and regulatory application support

Genetic Modification Clinical Research Information System (GeMCRIS) (www.gemcris.od.nih.gov/Contents/GC_HOME.asp)

Information resource and analytical tool that allows public users to access basic reports about gene transfer trials and develop specific queries (e.g., vector, transgene, medical condition)

Rare Diseases Clinical Research Network (RDCRN) (http://rarediseases.info.nih.gov/ASP/resources/extr_res.asp)

Program coordinated by the Office of Rare Diseases Research, National Center for Advancing Translational Science (NCATS), to support and facilitate research by creating consortia of research for related rare diseases with the goals of cost sharing, establishing uniform protocols, and collaborative clinical research

Clinical and Translational Science Award (CTSA) (www.ncats.nih.gov/research/cts/ctsa/about/about.html)

Program within the Division of Clinical Innovation, NCATS, provides funding to individual universities to increase the efficiency and speed of clinical and translational research across the country.

Foundations Funding Gene Transfer Research

Foundation Fighting Blindness (www.blindness.org/)

National Tay-Sachs & Allied Diseases Association, Inc. (www.ntsad.org/)

Muscular Dystrophy Association (http://mda.org/)

Batten Disease Support and Research Association (www.bdsra.org/)

Alliance for Cancer Gene Therapy (www.acgtfoundation.org/)