Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping

Prolonged expression of the CRISPR-Cas9 nuclease and gRNA from viral vectors may cause off-target mutagenesis and immunogenicity. Thus, a transient delivery system is needed for therapeutic genome editing applications. Here, we develop an extracellular nanovesicle-based ribonucleoprotein delivery sy...

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Detalles Bibliográficos
Autores principales: Gee, Peter, Lung, Mandy S. Y., Okuzaki, Yuya, Sasakawa, Noriko, Iguchi, Takahiro, Makita, Yukimasa, Hozumi, Hiroyuki, Miura, Yasutomo, Yang, Lucy F., Iwasaki, Mio, Wang, Xiou H., Waller, Matthew A., Shirai, Nanako, Abe, Yasuko O., Fujita, Yoko, Watanabe, Kei, Kagita, Akihiro, Iwabuchi, Kumiko A., Yasuda, Masahiko, Xu, Huaigeng, Noda, Takeshi, Komano, Jun, Sakurai, Hidetoshi, Inukai, Naoto, Hotta, Akitsu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7070030/
https://www.ncbi.nlm.nih.gov/pubmed/32170079
http://dx.doi.org/10.1038/s41467-020-14957-y

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7070030/
https://www.ncbi.nlm.nih.gov/pubmed/32170079
http://dx.doi.org/10.1038/s41467-020-14957-y

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