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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



Human Gene Therapy 24(4): 355-362



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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Accession: 053367077

Download citation: RISBibTeXText

PMID: 23517518

DOI: 10.1089/hum.2013.064


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