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Gene Therapy Scientific Interest Group

The Trans-NIH Gene Therapy Scientific Interest Group was developed to strengthen cross-disciplinary gene-therapy research at NIH. The diversity of gene therapy dictated the need to establish the Gene Therapy SIG to promote synergy across the intramural research program (IRP). For example, the broad clinical areas of interest are rare diseases, pediatric diseases, neurological diseases, infectious diseases, metabolic diseases, and cancer. The translational and regulatory aspects of gene therapy span the following areas of interest: target identification; preclinical research (including pharmacokinetics, pharmacodynamics, and toxicity); clinical trials; bioethics; and regulatory affairs. 

The goals of the Gene Therapy SIG are to:

  1. provide leadership, vision, and support to promote gene-therapy research within the IRP;
  2. share knowledge on gene-therapy research across institutions, disciplines, and NIH institutes and centers (ICs);
  3. collect, evaluate, and disseminate resources and opportunities for gene-therapy research at NIH;
  4. promote interactions, collaborations, and mentorship among intramural scientists and clinicians, and with extramural academic and industry researchers; and
  5. establish and implement a cohesive roadmap for gene-therapy research at NIH.

The Gene Therapy SIG members include program officers, program directors, and intramural investigators from different ICs. SIG activities will be coordinated by a steering committee that oversees the organization of meetings and educational offerings on developing and applying gene therapy within NIH. Extramural academic and industry researchers may be invited to attend as guests. A combination of face-to-face meetings and remote webinars will be held, featuring guest speakers to provide perspective, comments, and expert feedback. The steering committee will communicate the Gene Therapy SIG recommendations and suggestions to the Gene Therapy Taskforce, and vice versa.

Mailing List

To join the Gene Therapy Scientific Interest Group mailing list, please visit the Gene Therapy Scientific Interest Group Listserv home page, then click the “Subscribe or Unsubscribe” link in the right sidebar.



The page was last updated on Wednesday, December 1, 2021 - 4:11pm