Autor: |
O'Reilly M; Office of Biotechnology Activities, Office of Science Policy, Office of the Director, National Institutes of Health, Bethesda, MD 20892, USA., 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 |
Jazyk: |
angličtina |
Zdroj: |
Human gene therapy [Hum Gene Ther] 2013 Apr; Vol. 24 (4), pp. 355-62. |
DOI: |
10.1089/hum.2013.064 |
Abstrakt: |
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. |
Databáze: |
MEDLINE |
Externí odkaz: |
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