Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

The combination of Cas9, guide RNA and repair template DNA can induce precise gene editing and the correction of genetic diseases in adult mammals. However, clinical implementation of this technology requires safe and effective delivery of all of these components into the nuclei of the target tissue...

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Detalles Bibliográficos
Autores principales: Yin, Hao, Song, Chun-Qing, Dorkin, Joseph R, Zhu, Lihua J, Li, Yingxiang, Wu, Qiongqiong, Park, Angela, Yang, Junghoon, Suresh, Sneha, Bizhanova, Aizhan, Gupta, Ankit, Bolukbasi, Mehmet F, Walsh, Stephen, Bogorad, Roman L, Gao, Guangping, Weng, Zhiping, Dong, Yizhou, Koteliansky, Victor, Wolfe, Scot A, Langer, Robert, Xue, Wen, Anderson, Daniel G
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5423356/
https://www.ncbi.nlm.nih.gov/pubmed/26829318
http://dx.doi.org/10.1038/nbt.3471

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5423356/
https://www.ncbi.nlm.nih.gov/pubmed/26829318
http://dx.doi.org/10.1038/nbt.3471

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