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New Autism Protein Map Offers Path Toward Targeted Treatments, Doctors Say
Researchers identified 1,800 autism-linked protein interactions, and 87% were previously unseen, offering a blueprint for more targeted therapies.
On Thursday, University of California, San Francisco researchers published the largest molecular map of autism in the journal Science, revealing how genetic mutations rewire brain protein interactions.
With more than 250 genes linked to autism, finding treatments for individual genetic roots proved difficult, prompting researchers to target shared biological pathways instead.
Scientists used the artificial intelligence system AlphaFold to identify more than 1,800 protein interactions, 87% of which were previously unknown, analyzed within lab-grown brain organoids.
Study author Dr. Nevan Krogan confirmed three programs are developing new therapies using these insights, while Alison Singer, president of the Autism Science Foundation, noted this could address underlying biology for the 30% with profound autism.
While supporters describe this as a "watershed moment," Autism Speaks' chief science officer Dr. Andy Shih cautioned that translating these discoveries into clinical applications remains a long process requiring significant additional research.
Researchers in the US have made significant scientific progress in understanding the molecular mechanisms of autism, mapping the network for the first time.