論文

査読有り
2016年7月

Generation of a Nonhuman Primate Model of Severe Combined Immunodeficiency Using Highly Efficient Genome Editing

CELL STEM CELL
  • Kenya Sato
  • ,
  • Ryo Oiwa
  • ,
  • Wakako Kumita
  • ,
  • Rachel Henry
  • ,
  • Tetsushi Sakuma
  • ,
  • Ryoji Ito
  • ,
  • Ryoko Nozu
  • ,
  • Takashi Inoue
  • ,
  • Ikumi Katano
  • ,
  • Kengo Sato
  • ,
  • Norio Okahara
  • ,
  • Junko Okahara
  • ,
  • Yoshihisa Shimizu
  • ,
  • Masafumi Yamamoto
  • ,
  • Kisaburo Hanazawa
  • ,
  • Takao Kawakami
  • ,
  • Yoshie Kametani
  • ,
  • Ryuji Suzuki
  • ,
  • Takeshi Takahashi
  • ,
  • Edward J. Weinstein
  • ,
  • Takashi Yamamoto
  • ,
  • Yasubumi Sakakibara
  • ,
  • Sonoko Habu
  • ,
  • Jun-ichi Hata
  • ,
  • Hideyuki Okano
  • ,
  • Erika Sasaki

19
1
開始ページ
127
終了ページ
138
記述言語
英語
掲載種別
研究論文(学術雑誌)
DOI
10.1016/j.stem.2016.06.003
出版者・発行元
CELL PRESS

Recent advances in genome editing have facilitated the generation of nonhuman primate (NHP) models, with potential to unmask the complex biology of human disease not revealed by rodent models. However, their broader use is hindered by the challenges associated with generation of adult NHP models as well as the cost of their production. Here, we describe the generation of a marmoset model of severe combined immunodeficiency (SCID). This study optimized zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) to target interleukin-2 receptor subunit gamma (IL2RG) in pronuclear stage marmoset embryos. Nine of 21 neonates exhibited mutations in the IL2RG gene, concomitant with immunodeficiency, and three neonates have currently survived from 240 days to 1.8 years. Our approach demonstrates highly efficient production of founder NHP with SCID phenotypes, with promises of multiple preclinical and translational applications.

Web of Science ® 被引用回数 : 56

リンク情報
DOI
https://doi.org/10.1016/j.stem.2016.06.003
PubMed
https://www.ncbi.nlm.nih.gov/pubmed/27374787
Web of Science
https://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcAuth=JSTA_CEL&SrcApp=J_Gate_JST&DestLinkType=FullRecord&KeyUT=WOS:000381620500017&DestApp=WOS_CPL