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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Detalhes bibliográficos
Principais autores: Yin, Hao, Song, Chun-Qing, Suresh, Sneha, Wu, Qiongqiong, Walsh, Stephen C, Rhym, Luke Hyunsik, Mintzer, Esther, Bolukbasi, Mehmet Fatih, Zhu, Lihua Julie, Kauffman, Kevin John, Mou, Haiwei, Oberholzer, Alicia, Ding, Junmei, Kwan, Suet-Yan, Bogorad, Roman, Zatsepin, Timofei, Koteliansky, Victor, Wolfe, Scot A, Xue, Wen, Langer, Robert, Langer, Robert S, Anderson, Daniel Griffith
Outros Autores: Massachusetts Institute of Technology. Department of Chemical Engineering
Formato: Artigo
Idioma:English
Publicado em: Springer Science and Business Media LLC 2019
Acesso em linha:https://hdl.handle.net/1721.1/121981

Internet

https://hdl.handle.net/1721.1/121981

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