动物造模,单碱基突变,遗传性疾病动物模型 z-bio 2021-09-24 628

　　I learned from Jilin University that Lai Liangxue’s team from the School of Veterinary Medicine of Jilin University recently used a new single-base editing system to successfully implement single-base precise mutations in rabbits, and bred rabbits with genetic diseases such as albinism and progeria. This represents human distance. The era of gene therapy is one step closer.

　　Team member Li Zhanjun, PhD from the School of Animal Medicine of Jilin University, introduced that genetic diseases such as albinism and progeria are caused by single-base mutations in the genome. Because genetic diseases are caused by congenital genomic defects, traditional medicines are not effective for their treatment. Taking gene therapy methods, that is, directly repairing single-base mutations on the genome is the best treatment. The current gene therapy technology is still unable to meet the requirements of clinical treatment.

　　Lai Liangxue’s team used the latest new "base editors" (BE3 and ABE) used internationally to take the lead in the world to change single bases on model animal rabbits, accurately mimicking human single-base mutation genetic diseases The nonsense mutations, missense mutations, and RNA miscutting of the disease have successfully bred rabbits with diseases such as albinism, progeria, and gluteal muscles. Rabbits are closer to humans in terms of genetics, physiology and anatomy. Therefore, the results indicate that humans are one step closer to realizing gene therapy of this type of genetic disease.

　　The team is the first internationally to change a single base on a model animal rabbit, which stems from the experience accumulated over many years in the research of biomedicine and animal breeding using gene editing. Huang Xingxu, a gene editing expert and professor at Shanghai University of Science and Technology, believes that the study has confirmed the feasibility of using base editors for safe, precise, and efficient gene therapy. The results have been published in the internationally renowned journal "Nature-Communication", and the research has received special funding from the National Key Research and Development Program "Stem Cell and Transformation Research".

　　In the next step, Lai Liangxue’s team will apply the technological achievements to the molecular treatment of human genetic diseases as soon as possible, cooperate with other research units to carry out preclinical research on related diseases, and will continue to develop and optimize new gene editing systems for complex genetic diseases. Model construction and gene therapy.