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. 2013 Apr;24(4):355-62.
doi: 10.1089/hum.2013.064.

Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012

Marina O'Reilly  1 , Donald B Kohn, Jeffrey Bartlett, Janet Benson, Philip J Brooks, Barry J Byrne, Carlos Camozzi, Kenneth Cornetta, Ronald G Crystal, Yuman Fong, Linda Gargiulo, Rashmi Gopal-Srivastava, Katherine A High, Samuel G Jacobson, Robert C Jambou, Maureen Montgomery, Eugene Rosenthal, R Jude Samulski, Sonia I Skarlatos, Brian Sorrentino, James M Wilson, Yun Xie, Jacqueline Corrigan-Curay
Affiliations

Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012

Marina O'Reilly et al. Hum Gene Ther. 2013 Apr.

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.

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