Genome editing system shows promise for treating ASD

South China Morning Post

Chinese scientists have developed a genome editing system that successfully modified the DNA of mice with autism spectrum disorder (ASD) mutations. The treatment, delivered through a single injection, reduced ASD-associated behavior. Published in Nature Neuroscience, the study suggests potential treatment for ASD and other genetic neurodevelopmental disorders. The system, using a single-base editing method, restored protein levels in the brain and reversed behavioral abnormalities in mice. This breakthrough could lead to affordable individualized gene editing therapy for patients.


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