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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Библиографические подробности
Опубликовано в: :Nat Biotechnol
Главные авторы: Yin, Hao, Song, Chun-Qing, Suresh, Sneha, Wu, Qiongqiong, Walsh, Stephen, Rhym, Luke Hyunsik, Mintzer, Esther, Bolukbasi, Mehmet Fatih, Zhu, Lihua Julie, Kauffman, Kevin, Mou, Haiwei, Oberholzer, Alicia, Ding, Junmei, Kwan, Suet-Yan, Bogorad, Roman L, Zatsepin, Timofei, Koteliansky, Victor, Wolfe, Scot A, Xue, Wen, Langer, Robert, Anderson, Daniel G
Формат: Artigo
Язык:Inglês
Опубликовано: 2017
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Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC5901668/
https://ncbi.nlm.nih.gov/pubmed/29131148
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nbt.4005
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