Adeno-Associated Virus as a Vector for Liver-Directed Gene Therapy-Reference-Cited by-同舟云学术

Adeno-Associated Virus as a Vector for Liver-Directed Gene Therapy

Published:1998-12 Issue:12 Volume:72 Page:10222-10226
ISSN:0022-538X
Container-title:Journal of Virology
language:en
Short-container-title:J Virol

Author:

Xiao Weidong¹,Berta Scott C.¹,Lu Min Min¹,Moscioni A. David¹,Tazelaar John¹,Wilson James M.¹

Affiliation:

1. Institute for Human Gene Therapy and Departments of Molecular and Cellular Engineering and of Medicine, University of Pennsylvania, and the Wistar Institute, Philadelphia, Pennsylvania

Abstract

ABSTRACT Factors relevant to the successful application of adeno-associated virus (AAV) vectors for liver-directed gene therapy were evaluated. Vectors with different promoters driving expression of human α-1-antitrypsin (α-1AT) were injected into the portal circulation of immunodeficient mice. α-1AT expression was stable but dependent on the promoter. Southern analysis of liver DNA revealed approximately 0.1 to 2.0 provirus copies/diploid genome in presumed head-to-tail concatamers. In situ hybridization and immunohistochemical analysis revealed expression in approximately 5% of hepatocytes clustered in the pericentral region. These results support the use of AAV as a vector for diseases treatable by targeting of hepatocytes.

Publisher

American Society for Microbiology

Subject

Virology,Insect Science,Immunology,Microbiology

Link

https://journals.asm.org/doi/pdf/10.1128/JVI.72.12.10222-10226.1998

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