Скачать или смотреть Chinese Breakthrough: Gene Therapy Shows Promise in Autism Treatment| China news

Chinese scientists have achieved a significant milestone in gene therapy for autism by developing a genome editing system that successfully modified a mutated gene linked to autism in mice. The treatment, tested on mice, showed positive results in reducing ASD associated behavior. The researchers believe this treatment method could be used for other genetic neurodevelopmental disorders as well. To minimize unintended mutations, the Chinese scientists used a base editing system called AeCBE, which can work on individual DNA base pairs without creating any cuts. This approach is different from CRISPR based systems, which can lead to unintended mutations. The successful treatment of mice with ASD related mutations using base editing in the brain is a significant breakthrough in the field of gene therapy for autism. Researchers packaged their editing system on to a virus vector to cross the blood brain barrier. The system was administered to mutant mice through a single injection and successfully restored protein levels in the brain. The study provides guidance in using base editing to treat neurodevelopmental disorders. The team believes that with expansion of the technology and lower costs, individualized gene editing therapy could become feasible and affordable for patients in the near future.
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