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Open Access Methodology

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

Jun-Hyeok Choi1, Nam-Kyung Yu1, Gi-Chul Baek1, Joseph Bakes1, Daekwan Seo12, Hye Jin Nam14, Sung Hee Baek14, Chae-Seok Lim1, Yong-Seok Lee3 and Bong-Kiun Kaang1*

  • * Corresponding author: Bong-Kiun Kaang kaang@snu.ac.kr

  • † Equal contributors

Author Affiliations

1 Department of Biological Sciences, College of Natural Sciences, Seoul National University, Seoul 151-747, Korea

2 Center for RNA Research, Institute for Basic Science, Seoul, Korea

3 Department of Life Science, College of Natural Science, Chung-Ang University, Seoul 156-756, Korea

4 Department of Biological Sciences, Creative Research Initiatives Center for Chromatin Dynamics, Seoul National University, Seoul 151-742, South Korea

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Molecular Brain 2014, 7:17  doi:10.1186/1756-6606-7-17

Published: 11 March 2014

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.

Keywords:
Adeno-associated virus; WPRE; SV40 late polyadenylation signal sequence; Neuron