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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Nat Commun
Prif Awduron: 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
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Nature Publishing Group UK 2020
Pynciau:
Article
Mynediad Ar-lein: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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