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Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity
Adeno-associated virus (AAV) vector liver-directed gene therapy (GT) for hemophilia B (HB) is limited by a vector dose–dependent hepatotoxicity. Recently, this obstacle has been partially circumvented by the use of a hyperactive factor IX (FIX) variant, R338L (Padua), which has an 8-fold increased s...
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| Published in: | JCI Insight |
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| Main Authors: | , , , , |
| Format: | Artigo |
| Language: | Inglês |
| Published: |
American Society for Clinical Investigation
2019
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| Subjects: | |
| Online Access: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6675543/ https://ncbi.nlm.nih.gov/pubmed/31219805 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.128683 |
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