Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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AAV Hybrid Serotypes: Improved Vectors for Gene Delivery

Author(s): Vivian W. Choi, Douglas M. McCarty and R. Jude Samulski

Affiliation: 7119 Thurston Bowles CBᲸ, University of North Carolina at Chapel Hill, NC 27599, USA.

Abstract:

In recent years, significant efforts have been made on studying and engineering adeno-associated virus (AAV) capsid, in order to increase efficiency in targeting specific cell types that are non-permissive to wild type (wt) viruses and to improve efficacy in infecting only the cell type of interest. With our previous knowledge of the viral properties of the naturally occurring serotypes and the elucidation of their capsid structures, we can now generate capsid mutants, or hybrid serotypes, by various methods and strategies. In this review, we summarize the studies performed on AAV retargeting, and categorize the available hybrid serotypes to date, based on the type of modification: 1) transcapsidation, 2) adsorption of bi-specific antibody to capsid surface, 3) mosaic capsid, and 4) chimeric capsid. Not only these hybrid serotypes could achieve high efficiency of gene delivery to a specific targeted cell type, which can be better-tailored for a particular clinical application, but also serve as a tool for studying AAV biology such as receptor binding, trafficking and genome delivery into the nucleus.

Keywords: adeno associated virus, serotypes, capsids, tissue tropism

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Article Details

VOLUME: 5
ISSUE: 3
Page: [299 - 310]
Pages: 12
DOI: 10.2174/1566523054064968
Price: $58