Original paper
Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
Abstract
Efficient Cas9 genome editing in vivo is achieved without viral vectors using chemically modified single-guide RNAs. Efficient genome editing with Cas9–sgRNA in vivo has required the use of viral delivery systems, which have limitations for clinical applications. Translational efforts to develop other RNA therapeutics have shown that judicious chemical modification of RNAs can improve therapeutic efficacy by reducing susceptibility to nuclease...
Paper Details
Title
Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
Published Date
Nov 13, 2017
Journal
Volume
35
Issue
12
Pages
1179 - 1187
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Notes
History