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AAV-Mediated CRISPR/Cas Gene Editing of Retinal Cells In Vivo
Author(s) -
Sandy Hung,
Vicki Chrysostomou,
Li Fan,
Jeremiah K. H. Lim,
Jiang-Hui Wang,
Joseph E. Powell,
Leilei Tu,
Maciej Daniszewski,
Camden Lo,
Raymond C.B. Wong,
Jonathan G. Crowston,
Alice Pébay,
Anna E. King,
Bang V. Bui,
GueiSheung Liu,
Alex W. Hewitt
Publication year - 2016
Publication title -
investigative ophthalmology and visual science
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.935
H-Index - 218
eISSN - 1552-5783
pISSN - 0146-0404
DOI - 10.1167/iovs.16-19316
Subject(s) - crispr , biology , genome editing , yellow fluorescent protein , retinal , transgene , cas9 , adeno associated virus , subgenomic mrna , genetic enhancement , microbiology and biotechnology , gene , genetics , vector (molecular biology) , recombinant dna , biochemistry
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein (Cas) has recently been adapted to enable efficient editing of the mammalian genome, opening novel avenues for therapeutic intervention of inherited diseases. In seeking to disrupt yellow fluorescent protein (YFP) in a Thy1-YFP transgenic mouse, we assessed the feasibility of utilizing the adeno-associated virus 2 (AAV2) to deliver CRISPR/Cas for gene modification of retinal cells in vivo.

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