Title:
Non-human primate models in biomedical research

Erika Sasaki
Department Head
Department of Applied Developmental Biology,
Keio Advanced Research Center, Kio University, Sinjuku, Tokyo, Japan
Central Institute for Experimental Animals, Kawasaki, Kanagawa Japan

Abstract:
　Non-human primates offer excellent, precise preclinical study systems for assessing the safety and efficacy of new therapies and drugs. In particular, non-human primates are expected to be models for regenerative medicine using pluripotent stem cells and models of brain science to understand the molecular mechanisms of high cognitive function and the onset of nervous system diseases.
The common marmoset (Callithrix jacchus) is a useful experimental animal in biomedical research because of its similarity to humans, high reproductive efficiency, and easy handling. Given its prolificacy, the marmoset is suitable for producing genetically modified animals. Recent progress in transgenic technologies in non-human primates has enabled the generation of many human disease models. Furthermore, several pluripotent stem cells, including embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs), have been established in the marmoset and used in preclinical studies for regenerative medicine.
In many species, including the marmoset, targeted gene knock-out (KO)/knock-in (KI) animals cannot be achieved via traditional gene-targeting of ESCs or iPSCs as they lack the ability to contribute to the germline, although this is not the case in rodents. The recent development of innovative genome editing technologies such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALEN), and clustered regulatory interspaced short palindromic repeat (CRISPR)/Cas9 can resolve this issue, and it is now possible to generate target-gene KO animals without using ESCs. Recently, we performed marmoset IL2 receptor common γ chain gene knockout using ZFNs. This should be a useful model in haematology, cancer, or regenerative medicine research.