Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop, September 13, 2012

Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop, September 13, 2012 . Marina O'Reilly, 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, and Jacqueline Corrigan-Curay. Human Gene Therapy. April 2013, 24(4): 355-362. doi:10.1089/hum.2013.064.

Gene therapy has shown clinical efficacy for several rare diseases, using different approaches and vectors.