Nature Communications (Mar 2020)
Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
- 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
Affiliations
- Peter Gee
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Mandy S. Y. Lung
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Yuya Okuzaki
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Noriko Sasakawa
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Takahiro Iguchi
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Yukimasa Makita
- Takeda-CiRA Joint Program (T-CiRA)
- Hiroyuki Hozumi
- Takeda-CiRA Joint Program (T-CiRA)
- Yasutomo Miura
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Lucy F. Yang
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Mio Iwasaki
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Xiou H. Wang
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Matthew A. Waller
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Nanako Shirai
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Yasuko O. Abe
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Yoko Fujita
- Laboratory of Ultrastructural Virology, Institute for Frontier Life and Medical Sciences, Kyoto University
- Kei Watanabe
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Akihiro Kagita
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Kumiko A. Iwabuchi
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Masahiko Yasuda
- Pathology Analysis Center, Central Institute for Experimental Animals
- Huaigeng Xu
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Takeshi Noda
- Laboratory of Ultrastructural Virology, Institute for Frontier Life and Medical Sciences, Kyoto University
- Jun Komano
- Department of Clinical Laboratory, Nagoya Medical Center
- Hidetoshi Sakurai
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- Naoto Inukai
- Takeda-CiRA Joint Program (T-CiRA)
- Akitsu Hotta
- Center for iPS Cell Research and Application (CiRA), Kyoto University
- DOI
- https://doi.org/10.1038/s41467-020-14957-y
- Journal volume & issue
-
Vol. 11,
no. 1
pp. 1 – 18
Abstract
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.
