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Protocol
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

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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