title: 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.
authors.affiliation: Dimension Therapeutics , Cambridge, Massachusetts.
authors.forename: Lisa M
authors.initials: LM
authors.lastname: Kattenhorn
authors.affiliation: Dimension Therapeutics , Cambridge, Massachusetts.
authors.forename: Christopher H
authors.initials: CH
authors.lastname: Tipper
authors.affiliation: Dimension Therapeutics , Cambridge, Massachusetts.
authors.forename: Lorelei
authors.initials: L
authors.lastname: Stoica
authors.affiliation: Dimension Therapeutics , Cambridge, Massachusetts.
authors.forename: Deborah S
authors.initials: DS
authors.lastname: Geraghty
authors.affiliation: Dimension Therapeutics , Cambridge, Massachusetts.
authors.forename: Teresa L
authors.initials: TL
authors.lastname: Wright
authors.affiliation: Dimension Therapeutics , Cambridge, Massachusetts.
authors.forename: K Reed
authors.initials: KR
authors.lastname: Clark
authors.affiliation: Dimension Therapeutics , Cambridge, Massachusetts.
authors.forename: Samuel C
authors.initials: SC
authors.lastname: Wadsworth
mesh_terms: D000818:Animals; D000229:Dependovirus / Q000235:genetics*; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000008:administration & dosage*; D006801:Humans; D008107:Liver Diseases / Q000235:genetics* / Q000628:therapy*
keywords: AAV; clinical trials; gene therapy; hemophilia; liver; metabolic