Publication Type	Conference Paper
Authors	O'Reilly M, Kohn D, Bartlett J, Benson J, Brooks P, Byrne B, Camozzi C, Cornetta K, Crystal R, Fong Y, Gargiulo L, Gopal-Srivastava R, High K, Jacobson S, Jambou R, Montgomery M, Rosenthal E, Samulski R, Skarlatos S, Sorrentino B, Wilson J, Xie Y, Corrigan-Curay J
Journal	Hum Gene Ther
Volume	24
Issue	4
Pagination	355-62
Date Published	04/01/2013
ISSN	1557-7422
Keywords	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.
DOI	10.1089/hum.2013.064
PubMed ID	23517518
PubMed Central ID	PMC3631014