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 SorrentinoJames M. Wilson, Yun Xie, Jacqueline Corrigan-Curay

Research output: Contribution to journalReview article

14 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)355-362
Number of pages8
JournalHuman gene therapy
Volume24
Issue number4
DOIs
StatePublished - Apr 1 2013

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ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics

Cite this

O'Reilly, M., Kohn, D. B., Bartlett, J., Benson, J., Brooks, P. J., Byrne, B. J., Camozzi, C., Cornetta, K., Crystal, R. G., Fong, Y., Gargiulo, L., Gopal-Srivastava, R., High, K. A., Jacobson, S. G., Jambou, R. C., Montgomery, M., Rosenthal, E., Samulski, R. J., Skarlatos, S. I., ... Corrigan-Curay, J. (2013). Gene therapy for rare diseases: Summary of a National Institutes of Health Workshop, September 13, 2012. Human gene therapy, 24(4), 355-362. https://doi.org/10.1089/hum.2013.064