2016年7月
Generation of a Nonhuman Primate Model of Severe Combined Immunodeficiency Using Highly Efficient Genome Editing
CELL STEM CELL
- 巻
- 19
- 号
- 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.
- リンク情報
- ID情報
-
- DOI : 10.1016/j.stem.2016.06.003
- ISSN : 1934-5909
- eISSN : 1875-9777
- PubMed ID : 27374787
- Web of Science ID : WOS:000381620500017