Gene therapy for rare diseases: Summary of a National Institutes of Health Workshop, September 13, 2012 Article uri icon

Overview

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

Research

keywords

  • Article

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

  • 62

volume

  • 24

number

  • 4