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

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 inve...

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Detalhes bibliográficos
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2013
Assuntos:
Commentary
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3631014/
https://ncbi.nlm.nih.gov/pubmed/23517518
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2013.064
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