Adeno-Associated Virus Gene Therapy for Liver Disease

Hum Gene Ther. 2016 Dec;27(12):947-961. doi: 10.1089/hum.2016.160.


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.

Keywords: AAV; clinical trials; gene therapy; hemophilia; liver; metabolic.

Publication types

  • Review

MeSH terms

  • Animals
  • Dependovirus / genetics*
  • Genetic Therapy*
  • Genetic Vectors / administration & dosage*
  • Humans
  • Liver Diseases / genetics*
  • Liver Diseases / therapy*