期刊
CURRENT GENE THERAPY
卷 5, 期 3, 页码 299-310出版社
BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/1566523054064968
关键词
adeno associated virus; serotypes; capsids; tissue tropism
资金
- NHLBI NIH HHS [HL051818, P01 HL066973, HL066973, P01 HL051818] Funding Source: Medline
- NIAID NIH HHS [R01 AI048074, AI048074] Funding Source: Medline
- NIGMS NIH HHS [P01 GM059299] Funding Source: Medline
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.
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