Yunnan scientist gene edited "autistic monkey"

Yunnan scientist gene edited "autistic monkey"

June 06, 2017 Source: China Youth Daily

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The two little monkeys in the nursery box, a lively and active, full of interest in everything around them, touched this touch; one facing the wall, with fingers for a long time motionless, indifferent to the friendly gesture of the companion.

This is a video recently seen by the China Youth Daily and Zhongqing Online Journalist at the Key Laboratory of Primate Biomedicine in Yunnan Province.

The little monkey facing the wall, socially and cognitively, is a "self-closing monkey" edited by the lab using genetics, and its behavior is similar to that of human autism.

“This is a model of the Crab Monkey Reiter Syndrome (RTT) obtained by our team using TALEN technology. Through research and exploration of its treatment methods, it will bring hope to patients with autism.” Yunnan Zhongke Primate Chen Yongchang, a researcher at the Key Laboratory of Biomedical Sciences and a professor at the Institute of Translational Medicine of Kunming University of Science and Technology, said.

According to Chen Yongchang, Reiter syndrome is a neurodevelopmental disease belonging to the autism spectrum, which causes abortion of male fetuses during embryonic period; and sick girls show up after 6-18 months of birth. Loss of language ability, poor sleep quality, social and cognitive impairment, slow brain development, abnormal electrocardiogram, abnormal motor ability and other symptoms.

The cause of this disease is caused by a mutation in the MECP2 gene located on the X chromosome. But unfortunately, due to the lack of ideal animal models, humans have not yet found a cure for RTT.

“Although some scientists have used mouse models to conduct research, the mouse model is quite different from clinical patients, and it is difficult to promote the in-depth understanding of the pathogenesis and treatment methods.” Chen Yongchang said that non-human primates, such as Rhesus macaques and cynomolgus monkeys, which have a highly similar genetic background and brain structure, are the ideal experimental animals for brain development and neurological diseases.

In order to establish the RTT monkey model, researchers from Kunming University of Science and Technology, Yunnan Provincial Key Laboratory of Biomedical Sciences and Tongji University School of Medicine used the targeted gene editing method TALEN. After years of research, the monkey model of MECP2 gene was successfully edited. . This is the first time that Chinese scientists have used genetic editing to produce the Reiter syndrome monkey model. This research is supported by the National Key Research and Development Program stem cell and transformation research project.

"Gene editing technology is the human 'editing' of the target gene, knocking out and adding specific DNA fragments, so that it can achieve the purpose of human design." Chen Yongchang said.

According to long-term observation, the small monkeys with the gene mutation MECP2 showed a series of pathological and behavioral characteristics very similar to those of autistic patients. For example, male fetuses will abort during embryonic period, females have sleep disorders, active social reduction, increased stereotypes, brain development abnormalities, etc.; primates have advanced cognitive ability eye tracking models similar to humans.

On May 18th, the international top journal "Cell" published the research results of Yunnan Zhongke Primate Biomedical Key Laboratory using TALEN technology to carry out the model of cynomolgus monkey Reiter syndrome.

"Using non-human primates to systematically study the behavioral patterns, pathogenesis and mechanisms of RTTs not only proves that non-human primates have an incomparable advantage over rodents in neurological disease research, but also deeper into the future. The research and treatment of the pathogenesis of RTT will have a profound impact." Ji Weizhi, Distinguished Professor of Kunming University of Science and Technology, and Director of the Yunnan Key Laboratory of Biomedical Animals.

It is worth mentioning that as the leader of the research team, Ji Weizhi, former director of the Kunming Institute of Zoology of the Chinese Academy of Sciences, pioneered the study of primate conservation biology, reproductive biology and stem cell biology in China. Ji Weizhi's series of research results play an important role in the internationalization of Chinese primate research and rank among the world's first.

It is reported that at present, the research team is discussing cooperation with the First People's Hospital of Yunnan Province, preparing to conduct clinical trials for the benefit of autistic patients. (Zhang Wenling)

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