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.

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 Gee^1,2,3, Mandy S.Y. Lung¹, Yuya Okuzaki¹, Noriko Sasakawa¹, Takahiro Iguchi¹, Yukimasa Makita^3,4, Hiroyuki Hozumi^3,4, 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. Iwabuchi^1,3, Masahiko Yasuda⁶, Huaigeng Xu¹, Takeshi Noda⁵, Jun Komano^7,8, Hidetoshi Sakurai¹, Naoto Inukai^3,4, and Akitsu Hotta^1,2,3
• 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