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

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Title
Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons
Author(s)
Jun-Hyeok Choi; Nam-Kyung Yu; Gi-Chul Baek; Joseph Bakes; Daekwan Seo; Hye Jin Nam; Sung Hee Baek; Chae-Seok Lim; Yong-Seok Lee; Bong-Kiun Kaang
Publication Date
2014-03
Journal
MOLECULAR BRAIN, v.7, no., pp.17 -
Publisher
BIOMED CENTRAL LTD
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.
URI
http://pr.ibs.re.kr/handle/8788114/1097
DOI
10.1186/1756-6606-7-17
ISSN
1756-6606
Appears in Collections:
Center for RNA Research(RNA 연구단) > Journal Papers (저널논문)
Files in This Item:
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