The reporter learned from Jilin University that the team of Lai Liangxue of the College of Animal Medicine of Jilin University recently succeeded in realizing single-base mutations in rabbits using a novel single-base editing system to breed rabbits with hereditary diseases such as albinism and premature aging, which represent humans. A step closer to the era of gene therapy.
Li Zhanjun, a team member and a doctor of the College of Animal Medicine of Jilin University, said that hereditary diseases such as albinism and premature aging are caused by single-base mutations in the genome. Because the cause of hereditary diseases is congenital genomic defects, traditional drugs are ineffective for their treatment. Gene therapy is the best treatment for directly repairing single-base mutations on the genome. The current gene therapy techniques are still unable to meet the requirements of clinical treatment.
The Lai Liangxue team used the new "base editor" (BE3 and ABE), which is the latest in the world, to take the lead in changing the single base in the model animal rabbits in the world, accurately simulating the human single base mutation genetic disease. The non-sense mutation, the missense mutation and the RNA miscutting successfully cultivated rabbits with disease models such as albinism, premature aging, and double muscle buttocks. Rabbits are genetic, physiological, and anatomically closer to humans, so the results mark a step closer to achieving genetic therapy for this type of hereditary disease.
The team is the first in the world to change a single base on a model animal rabbit, stemming from years of experience in biomedical and animal breeding research using genetic editing. Huang Xunxu, a genetic editing expert and professor at Shanghai University of Science and Technology, believes that the study confirms the feasibility of using a base editor for safe, accurate, and efficient gene therapy. The results have been published in the internationally renowned journal Nature-Communication, which received special funding from the National Key Research and Development Program “Stem Cell and Transformation Researchâ€.
In the next step, Lai Liangxue team will apply the technical results 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 continue to develop and optimize new gene editing systems for complex genetic diseases. Model construction and gene therapy. (Reporter Meng Hanqi Gao Nan)
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