AI-guided drug discovery forges a novel therapeutic path for patients with Rett syndrome

Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are born with it and much fewer boys. It is caused by mutations in the MeCP2 gene on the X chromosome, leading to a spectrum of cognitive and physical impairments, including repetitive hand motions, speech difficulties, and seizures.

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