期刊
JOURNAL OF INTERNAL MEDICINE
卷 280, 期 3, 页码 246-251出版社
WILEY
DOI: 10.1111/joim.12469
关键词
CRISPR; Cas9; disease model; genome editing; nonhuman primate; TALENs
资金
- National Basic Research Program of China [2012CBA01300, 2014CB560701]
- National High Technology Research and Development Program of China [2012AA020701]
- National Natural Science Foundation of China [U1302227, 31271599, 31571534]
- Yunnan Innovation Talents of Science and Technology [2012HA013, 2013HB133]
- Basic Research Project of Yunnan Province [2015FA037]
Nonhuman primates (NHPs) are superior than rodents to be animal models for the study of human diseases, due to their similarities in terms of genetics, physiology, developmental biology, social behaviour and cognition. Transgenic animals have become a key tool in functional genomics to generate models for human diseases and validate new drugs. However, until now, progress in the field of transgenic NHPs has been slow because of technological limitations. Many human diseases, including neurodegenerative disorders, are caused by mutations in endogenous genes. Fortunately, recent developments in precision gene editing have led to the generation of NHP models for human diseases. Since 2014, there have been several reports of the generation of monkey models using transcription activator-like endonucleases (TALENs) or clustered regularly interspaced short palindromic repeats (CRISPR/Cas9); some of these NHP models showed symptoms that were much closer to those of human diseases than have been seen previously in mouse models. No off-targeting was observed in the NHP models, and multiple gene knockout and biallelic mutants were feasible with low efficiency. These findings suggest that there are many possibilities to establish NHP models for human diseases that can mimic human diseases more faithfully than rodent models.
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