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 & 3 others James M. Wilson, Yun Xie, Jacqueline Corrigan-Curay

Research output: Contribution to journalArticle

14 Citations (Scopus)

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
Pages (from-to)355-362
Number of pages8
JournalHuman Gene Therapy
Volume24
Issue number4
DOIs
StatePublished - Apr 1 2013

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National Institutes of Health (U.S.)
Rare Diseases
Genetic Therapy
Education
Information Dissemination
Health Resources
United States Food and Drug Administration
Biotechnology
Research Personnel
Research

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., ... 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

Gene therapy for rare diseases : Summary of a National Institutes of Health Workshop, September 13, 2012. / O'Reilly, Marina; Kohn, Donald B.; Bartlett, Jeffrey; Benson, Janet; Brooks, Philip J.; Byrne, Barry J.; Camozzi, Carlos; Cornetta, Kenneth; Crystal, Ronald G.; Fong, Yuman; Gargiulo, Linda; Gopal-Srivastava, Rashmi; High, Katherine A.; Jacobson, Samuel G.; Jambou, Robert C.; Montgomery, Maureen; Rosenthal, Eugene; Samulski, R. Jude; Skarlatos, Sonia I.; Sorrentino, Brian; Wilson, James M.; Xie, Yun; Corrigan-Curay, Jacqueline.

In: Human Gene Therapy, Vol. 24, No. 4, 01.04.2013, p. 355-362.

Research output: Contribution to journalArticle

O'Reilly, M, Kohn, DB, Bartlett, J, Benson, J, Brooks, PJ, Byrne, BJ, Camozzi, C, Cornetta, K, Crystal, RG, Fong, Y, Gargiulo, L, Gopal-Srivastava, R, High, KA, Jacobson, SG, Jambou, RC, Montgomery, M, Rosenthal, E, Samulski, RJ, Skarlatos, SI, Sorrentino, B, Wilson, JM, Xie, Y & Corrigan-Curay, J 2013, 'Gene therapy for rare diseases: Summary of a National Institutes of Health Workshop, September 13, 2012', Human Gene Therapy, vol. 24, no. 4, pp. 355-362. https://doi.org/10.1089/hum.2013.064
O'Reilly, Marina ; Kohn, Donald B. ; Bartlett, Jeffrey ; Benson, Janet ; Brooks, Philip J. ; Byrne, Barry J. ; Camozzi, Carlos ; Cornetta, Kenneth ; Crystal, Ronald G. ; Fong, Yuman ; Gargiulo, Linda ; Gopal-Srivastava, Rashmi ; High, Katherine A. ; Jacobson, Samuel G. ; Jambou, Robert C. ; Montgomery, Maureen ; Rosenthal, Eugene ; Samulski, R. Jude ; Skarlatos, Sonia I. ; Sorrentino, Brian ; Wilson, James M. ; Xie, Yun ; Corrigan-Curay, Jacqueline. / Gene therapy for rare diseases : Summary of a National Institutes of Health Workshop, September 13, 2012. In: Human Gene Therapy. 2013 ; Vol. 24, No. 4. pp. 355-362.
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