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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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Main Authors: | , , , , , , , , , , , , , , , , |
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Format: | Artigo |
Sprog: | Inglês |
Udgivet: |
American Society of Hematology
2010
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Fag: | |
Online adgang: | 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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