Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models
Abstract Background Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia. However, these pivotal investigations have also identified several challenges. For example, high vector doses are oft...
Main Authors: | , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
BMC
2017-05-01
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Series: | Journal of Translational Medicine |
Subjects: | |
Online Access: | http://link.springer.com/article/10.1186/s12967-017-1200-1 |