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

Cited 58 time in Web of Science Cited 68 time in Scopus
Authors
Choi, Jun-Hyeok; Yu, Nam-Kyung; Baek, Gi-Chul; Bakes, Joseph; Seo, Daekwan; Nam, Hye Jin; Baek, Sung Hee; Lim, Chae-Seok; Lee, Yong-Seok; Kaang, Bong-Kiun
Issue Date
2014-03-11
Publisher
BioMed Central
Citation
Molecular Brain, 7(1):17
Keywords
Adeno-associated virusWPRESV40 late polyadenylation signal sequenceNeuron
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.
Language
English
URI
https://hdl.handle.net/10371/109817
DOI
https://doi.org/10.1186/1756-6606-7-17
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College of Natural Sciences (자연과학대학)Dept. of Biological Sciences (생명과학부)Journal Papers (저널논문_생명과학부)
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