Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models

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...

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Bibliographic Details
Main Authors: David M. Markusic, Timothy C. Nichols, Elizabeth P. Merricks, Brett Palaschak, Irene Zolotukhin, Damien Marsic, Sergei Zolotukhin, Arun Srivastava, Roland W. Herzog
Format: Article
Language:English
Published: BMC 2017-05-01
Series:Journal of Translational Medicine
Subjects:
AAV
Gene therapy
Hemophilia B
Factor IX
Capsid
Online Access:http://link.springer.com/article/10.1186/s12967-017-1200-1

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