Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
Abstract
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 degradation. Guided by the structure of the Cas9-sgRNA complex, we identify regions of sgRNA that can be modified...
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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