Exosome-associated SUMOylation mutant AAV demonstrates improved ocular gene transfer efficiency in vivo.
Virus Research(2020)
摘要
•A novel combination of an exosome associated with SUMOylation capsid-site engineered AAV vector has been tested for its ocular gene transfer potential.•Exosome -associated vectors show enhanced transduction in human retinal cells in vitro and in C57BL6/J mice in vivo after intravitreal gene transfer.•Our data highlights the translational potential of exosome mediated delivery of engineered vectors to enhance therapeutic gene expression in retinal cells.
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关键词
AAV2,GFP,vgs,LCA2,RPE65,Exo,RIPA,BCA,TBS,HeLa,ARPE19,h.FIX
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