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

Adeno-Associated Virus Gene Therapy for Liver Disease

Lisa M Kattenhorn  1 , Christopher H Tipper  1 , Lorelei Stoica  1 , Deborah S Geraghty  1 , Teresa L Wright  1 , K Reed Clark  1 , Samuel C Wadsworth  1
Affiliations
Review

Adeno-Associated Virus Gene Therapy for Liver Disease

Lisa M Kattenhorn et al. Hum Gene Ther. 2016 Dec.

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.

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

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Conflict of interest statement

Author Disclosure The authors declare that they have no potential competing financial interests. K.C. and S.W. are shareholders in Dimension Therapeutics. L.K., C.T., L.S., D.G., T.W., K.C., and S.W. are employees of Dimension Therapeutics.

Figures

<b>Figure 1.</b>
Figure 1.
Architecture of the liver sinusoid. Liver sinusoids (S) are lined by fenestrated endothelia (EC) and interspersed Kupffer cells (KC), the resident macrophages of the liver. Stellate cells (SC), the major producers of liver ECM, are located inside the narrow space of Disse (D), which is formed by the sinusoidal cell layer and cords of hepatocytes (H). Figure originally published in Frevert U, Engelmann S, Zougbédé S, et al. Intravital observation of plasmodium berghei sporozoite infection of the liver. PLoS Biol 2005;3:e192. DOI: 10.1371/journal.pbio.0030192.
See this image and copyright information in PMC

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