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Nature Publishes Research Results from Shanghai Jiao Tong University: A New Breakthrough in Gene Therapy for Autism
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Author：小编
Autism spectrum disorder (ASD) is associated with hundreds of genetic variations, with mutations in the CHD3 gene closely linked to core symptoms such as social impairments and repetitive behaviors. Traditional interventions primarily rely on behavioral therapy, which fails to address the underlying causes. A collaborative team from Shanghai Jiao Tong University utilized a novel base-editing technique (non-cleaving CRISPR) to deliver editing tools into the brains of mice and monkeys via AAV viral vectors, precisely correcting single-nucleotide mutations in the CHD3 gene.
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