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Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons.


ABSTRACT: Adeno-associated virus (AAV) vectors can deliver transgenes to diverse cell types and are therefore useful for basic research and gene therapy. Although AAV has many advantages over other viral vectors, its relatively small packaging capacity limits its use for delivering large genes. The available transgene size is further limited by the existence of additional elements in the expression cassette without which the gene expression level becomes much lower. By using alternative combinations of shorter elements, we generated a series of AAV expression cassettes and systematically evaluated their expression efficiency in neurons to maximize the transgene size available within the AAV packaging capacity while not compromising the transgene expression. We found that the newly developed smaller expression cassette shows comparable expression efficiency with an efficient vector generally used for strong gene expression. This new expression cassette will allow us to package larger transgenes without compromising expression efficiency.

SUBMITTER: Choi JH 

PROVIDER: S-EPMC3975461 | biostudies-literature | 2014

REPOSITORIES: biostudies-literature

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Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons.

Choi Jun-Hyeok JH   Yu Nam-Kyung NK   Baek Gi-Chul GC   Bakes Joseph J   Seo Daekwan D   Nam Hye Jin HJ   Baek Sung Hee SH   Lim Chae-Seok CS   Lee Yong-Seok YS   Kaang Bong-Kiun BK  

Molecular brain 20140311


Adeno-associated virus (AAV) vectors can deliver transgenes to diverse cell types and are therefore useful for basic research and gene therapy. Although AAV has many advantages over other viral vectors, its relatively small packaging capacity limits its use for delivering large genes. The available transgene size is further limited by the existence of additional elements in the expression cassette without which the gene expression level becomes much lower. By using alternative combinations of sh  ...[more]

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