Adeno-Associated Virus Gene Therapy for Liver Disease.

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.

SUBMITTER: Kattenhorn LM 

PROVIDER: S-EPMC5177998 | biostudies-literature | 2016 Dec

REPOSITORIES: biostudies-literature