Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

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 reduc...

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Main Authors: Yin, Hao (Author), Song, Chun-Qing (Author), Suresh, Sneha (Author), Wu, Qiongqiong (Author), Walsh, Stephen C (Author), Rhym, Luke Hyunsik (Author), Mintzer, Esther (Author), Bolukbasi, Mehmet Fatih (Author), Zhu, Lihua Julie (Author), Kauffman, Kevin John (Author), Mou, Haiwei (Author), Oberholzer, Alicia (Author), Ding, Junmei (Author), Kwan, Suet-Yan (Author), Bogorad, Roman (Author), Zatsepin, Timofei (Author), Koteliansky, Victor (Author), Wolfe, Scot A (Author), Xue, Wen (Author), Langer, Robert (Author), Langer, Robert S (Author), Anderson, Daniel Griffith (Author)
Other Authors: Massachusetts Institute of Technology. Department of Chemical Engineering (Contributor), Harvard University- (Contributor), Massachusetts Institute of Technology. Institute for Medical Engineering & Science (Contributor), Massachusetts Institute of Technology. Department of Biology (Contributor), Koch Institute for Integrative Cancer Research at MIT (Contributor)
Format: Article
Language:English
Published: Springer Science and Business Media LLC, 2019-08-12T15:29:27Z.
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