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Alternative Strategies for Gene Therapy of Hemophilia

Hemophilia A and B are monogenic disorders that were felt to be ideal targets for initiation of gene therapy. Although the first hemophilia gene therapy trial has been over 10 years ago, few trials are currently actively recruiting. Although preclinical studies in animals were promising, levels achi...

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Detalhes bibliográficos
Main Authors: Montgomery, Robert R., Shi, Qizhen
Formato: Artigo
Idioma:Inglês
Publicado em: 2010
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3383974/
https://ncbi.nlm.nih.gov/pubmed/21239794
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/asheducation-2010.1.197
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