Open Access
- © Christopher H Tipper
- , et al.
- 2022
Adeno-Associated Virus Gene Therapy for Liver Disease
- Christopher H Tipper 1
- Deborah S Geraghty 1
- Lisa M Kattenhorn 1
- Lorelei Stoica 1
- Reed Clark 1
- Samuel C Wadsworth 1
- Teresa L Wright 1
- 1 - na
Jun 28, 2022
Abstract
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 other liver disorders. This review focuses on liver-directed AAV gene therapy, including historic context, current challenges, and future developments.
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