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Article

Adeno-Associated Virus Gene Therapy for Liver Disease

29 Nov 2016

Abstract excerpt

The field of adeno-associated virus (AAV) gene therapy has progressed rapidly over the past decade, with the advent of novel capsid serotype and organ-specific promoters, and an increasing understanding of the immune response to AAV administration. In particular, liver-directed therapy has made remarkable strides, with a number of clinical trials currently planned and ongoing in hemophilia A and B, as well as...

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PMID: 27897038
DOI: 10.1089/hum.2016.160

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