Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Conference Paper uri icon

Overview

MeSH

  • Clinical Trials as Topic
  • Genetic Therapy
  • Humans
  • National Institutes of Health (U.S.)
  • Translational Medical Research
  • United States
  • United States Food and Drug Administration

MeSH Major

  • Rare Diseases

abstract

  • Gene therapy has shown clinical efficacy for several rare diseases, using different approaches and vectors. The Gene Therapy for Rare Diseases workshop, sponsored by the National Institutes of Health (NIH) Office of Biotechnology Activities and Office of Rare Diseases Research, brought together investigators from different disciplines to discuss the challenges and opportunities for advancing the field including means for enhancing data sharing for preclinical and clinical studies, development and utilization of available NIH resources, and interactions with the U.S. Food and Drug Administration.

publication date

  • April 2013

has subject area

  • Clinical Trials as Topic
  • Genetic Therapy
  • Humans
  • National Institutes of Health (U.S.)
  • Rare Diseases
  • Translational Medical Research
  • United States
  • United States Food and Drug Administration

Research

keywords

  • Congresses

Identity

Language

  • eng

PubMed Central ID

  • PMC3631014

Digital Object Identifier (DOI)

  • 10.1089/hum.2013.064

PubMed ID

  • 23517518

Additional Document Info

start page

  • 355

end page

  • 362

volume

  • 24

number

  • 4