The world's first Huntington's disease gene has been struck into a pig.

In the early hours of March 30, Beijing time, the international authoritative academic journal Cell published a major achievement in biology online: the international research team led by Guangdong scientists spent four years, the first use of gene editing technology (CRISPR/Cas9) and somatic cell nuclear transplantation technology, successfully bred the world's first Huntington's disease gene knockinto pigs, accurately simulated human neurodegenerative diseases.
the results were jointly completed by Li Xiaojiang, Professor of Guangdong, Hong Kong and Macao Institute of Central Neuroregeneration, Researcher Lai Liangxue of the Guangzhou Institute of Biomedicine and Health Of chinese Academy of Sciences, and Li Shihua, Professor of Emory University in the United States.
" In recent years, China has made breakthrough achievements in the research of gene-edited monkeys and cloned monkeys.
" Professor Yan Gang, a member of the Chinese Academy of Sciences, said after learning of the publication of the results that the Chinese scientists have made great progress in the study of disease models of pigs, indicating that China has been in the forefront of the research of large animal models in the world, and will greatly promote the innovation and development of China's biopharmaceutical industry.
it is understood that Li Xiaojiang and Li Shihua successfully established the world's first genetically modified Huntington's disease monkey model in 2008 with partners at Emory University in the United States, and in 2010, they worked with Lai Liangxuan to build the first model of a pig with a genetically modified Huntington's disease.
in an effort to build animal models that could more accurately simulate neurodegenerative diseases, in 2013 the two teams worked together again to try to insert the human mutation's Huntington gene into the expression box of the endogenous Huntington's gene in pigs.
after 4 years of efforts, researchers used gene editing (CRISPR/Cas9) technology to accurately insert the human mutation huntington gene, i.e. human exon 1 contains 150 CAG repeat sequences into the htT endogenous gene of pigs, use fibroblasts to screen positive cloned cells, and through somatic cell nuclear transplantation technology, successfully bred Huntington's disease gene into the pig model, the first time in the world to establish a similar to the neurodegenerative gene mutation.
studies have shown that the model not only simulates the typical pathological characteristics of selective death of medium-sized echidna neurons in the brain striatum in Huntington's disease patients, but also shows "dance-like" behavioral abnormalities similar to Huntington's disease on behavioral phenotypes.
more important, these pathological features and abnormal behavior can be passed on steadily to future generations.
the world's first Huntington's disease gene knocked into the pig model, in the simulation of neuropathological changes in patients, especially brain disease, large animal models than small animal models have an advantage.
" Lai Liangxuan told China Science Daily that the study provides a stable and reliable animal model for the development of new methods for the treatment of Huntington's disease, as well as a technical model and theoretical basis for the development of large animal models of other neurodegenerative diseases. Yang Xiangdong, a professor at the University of California, Los Angeles,
who has been studying Huntington's disease for many years, points out that the creation of the Huntington's disease gene is a landmark discovery in the field of neurodegenerative disease research, giving scientists a deeper understanding of the mechanisms of nerve cell death and finding effective treatments.
Source: Science.com