Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

Muscle represents an important tissue target for adeno-associated viral (AAV) vector-mediated gene transfer of the factor IX (FIX) gene in hemophilia B (HB) subjects with advanced liver disease. Previous studies of direct intramuscular administration of an AAV-FIX vector in humans showed limited eff...

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Detalhes bibliográficos
Main Authors: Arruda, Valder R., Stedman, Hansell H., Haurigot, Virginia, Buchlis, George, Baila, Stefano, Favaro, Patricia, Chen, Yifeng, Franck, Helen G., Zhou, Shangzhen, Wright, J. Fraser, Couto, Linda B., Jiang, Haiyan, Pierce, Glenn F., Bellinger, Dwight A., Mingozzi, Federico, Nichols, Timothy C., High, Katherine A.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2010
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2890180/
https://ncbi.nlm.nih.gov/pubmed/20335222
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2009-12-261156
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