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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מידע ביבליוגרפי
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.
פורמט: Artigo
שפה:Inglês
יצא לאור: American Society of Hematology 2010
נושאים:
Gene Therapy
גישה מקוונת: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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