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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Publicado en:Nat Commun
Main Authors: 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: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group UK 2020
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7070030/
https://ncbi.nlm.nih.gov/pubmed/32170079
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-020-14957-y
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