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An evolved adeno-associated viral variant enhances gene delivery and gene targeting in neural stem cells

 Jae-Hyung Jang  ;  James T Koerber  ;  Jung-Suk Kim  ;  Prashanth Asuri  ;  Tandis Vazin  ;  Melissa Bartel  ;  Albert Keung  ;  Inchan Kwon  ;  Kook In Park  ;  David V Schaffer 
 MOLECULAR THERAPY, Vol.19(4) : 667-675, 2011 
Journal Title
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Animals ; Cells, Cultured ; Chromatography ; Dependovirus/genetics* ; Female ; Fluorescent Antibody Technique ; Gene Transfer Techniques ; Neural Stem Cells/virology* ; Polymerase Chain Reaction ; Rats ; Rats, Inbred F344
Gene delivery to, and gene targeting in, stem cells would be a highly enabling technology for basic science and biomedical application. Adeno-associated viral (AAV) vectors have demonstrated the capacity for efficient delivery to numerous cells, but their application to stem cells has been limited by low transduction efficiency. Due to their considerable advantages, however, engineering AAV delivery systems to enhance gene delivery to stem cells may have an impact in stem cell biology and therapy. Therefore, using several diverse AAV capsid libraries-including randomly mutagenized, DNA shuffled, and random peptide insertion variants-we applied directed evolution to create a "designer" AAV vector with enhanced delivery efficiency for neural stem cells (NSCs). A novel AAV variant, carrying an insertion of a selected peptide sequence on the surface of the threefold spike within the heparin-binding site, emerged from this evolution. Importantly, this evolved AAV variant mediated efficient gene delivery to rat, mouse, and human NSCs, as well as efficient gene targeting within adult NSCs, and it is thus promising for applications ranging from basic stem cell biology to clinical translation.
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1. College of Medicine (의과대학) > Dept. of Pediatrics (소아청소년과학교실) > 1. Journal Papers
Yonsei Authors
Park, Kook In(박국인) ORCID logo https://orcid.org/0000-0001-8499-9293
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