Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models
Abstract
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 often used for hepatic gene transfer, and cytotoxic T lymphocyte responses against viral capsid may occur. Therefore, achieving therapy at reduced vector doses and other...
Paper Details
Title
Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models
Published Date
May 1, 2017
Volume
15
Issue
1
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