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The research publication of the in vivo genome editing project for congenital muscular dystrophy in T-CiRA Joint Program were published online in Nature Communications on March 13th 2020.

March 17, 2020

The research publication of the in vivo genome editing project for congenital muscular dystrophy in T-CiRA Joint Program were published online in Nature Communications on March 13th 2020.
https://www.nature.com/articles/s41467-020-14957-y

CiRA press release: https://www.cira.kyoto-u.ac.jp/e/pressrelease/news/200313-190000.html

Paper Details

  • Journal: Nature Communications
  • Title: Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
  • Authors: Peter Gee1,2,3, Mandy S.Y. Lung1, Yuya Okuzaki1, Noriko Sasakawa1, Takahiro Iguchi1, Yukimasa Makita3,4, Hiroyuki Hozumi3,4, Yasutomo Miura1, Lucy F. Yang1, Mio Iwasaki1, Xiou H. Wang1, Matthew A. Waller1, Nanako Shirai1, Yasuko O. Abe1, Yoko Fujita5, Kei Watanabe1, Akihiro Kagita1, Kumiko A. Iwabuchi1,3, Masahiko Yasuda6, Huaigeng Xu1, Takeshi Noda5, Jun Komano7,8, Hidetoshi Sakurai1, Naoto Inukai3,4, and Akitsu Hotta1,2,3
  • Author Affiliations:

1 Center for iPS Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan

2 Institute for Integrated Cell-Material Sciences (iCeMS), Kyoto University, Kyoto, Japan

3 Takeda-CiRA Joint Program (T-CiRA), Kanagawa, Japan

4 T-CiRA Discovery, Takeda Pharmaceutical Company Limited, Kanagawa, Japan

5 Laboratory of Ultrastructural Virology, Institute for Frontier Life and Medical Sciences, Kyoto University, Kyoto, Japan

6 Pathology Analysis Center, Central Institute for Experimental Animals, Kanagawa, Japan

7 Department of Clinical Laboratory, Nagoya Medical Center, Nagoya, Japan

8 Present address: Department of Infection Control, Osaka University of Pharmaceutical Sciences, Osaka, Japan