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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Xehetasun bibliografikoak
Argitaratua izan da:Nat Commun
Egile Nagusiak: 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
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Nature Publishing Group UK 2020
Gaiak:
Article
Sarrera elektronikoa: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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