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

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

Expression of Cas9 and gRNA from viral vectors in vivo may cause off-target activity. Here the authors present NanoMEDIC, which uses nanovesicles to transiently deliver editing machinery to hard-to-transfect cells.

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Autores principales: Peter Gee, Mandy S. Y. Lung, Yuya Okuzaki, Noriko Sasakawa, Takahiro Iguchi, Yukimasa Makita, Hiroyuki Hozumi, Yasutomo Miura, Lucy F. Yang, Mio Iwasaki, Xiou H. Wang, Matthew A. Waller, Nanako Shirai, Yasuko O. Abe, Yoko Fujita, Kei Watanabe, Akihiro Kagita, Kumiko A. Iwabuchi, Masahiko Yasuda, Huaigeng Xu, Takeshi Noda, Jun Komano, Hidetoshi Sakurai, Naoto Inukai, Akitsu Hotta
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2020
Materias:
Science
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Acceso en línea:https://doaj.org/article/4d0fe2ef9a604f0fb6865d1e08b1ed50
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