UCSF QBI Awarded $46M to Translate Landmark Autism Map Into Precision Therapies

UCSF's Quantitative Biosciences Institute (QBI), in collaboration with UCSF's Department of Psychiatry and Behavioral Sciences, has been awarded $46 million in grant funding from Aligning Research to Impact Autism (ARIA). This significant investment marks one of the largest in the QBI's history and builds upon a decade of collaborative research, known as the Psychiatric Cell Map Initiative (PCMI).

The purpose of this funding is to translate the landmark autism study, published in Science, into precision therapies for autism spectrum disorder (ASD). The study, led by scientists at QBI and UCSF, presents a molecular interaction map of autism, revealing how hundreds of genes and mutations converge within shared protein networks.

The funding will support QBI's work in ARIA's Protein-Protein Interactions Hub, one of six research hubs in ARIA's scientific ecosystem. This hub focuses on mapping disrupted protein complexes and developing small molecules to modulate their activity. The researchers used affinity purification-mass spectrometry to map proteins encoded by 100 autism-linked genes, discovering over 1,800 previously unreported protein interactions.

By integrating these interaction maps with structural modeling predictions and functional studies in cell models, the scientists pinpointed exactly where mutations disrupt protein interfaces. The findings show that distinct mutations in different autism genes can disrupt the same protein complexes, pointing to potential therapeutic targets that could treat multiple forms of autism simultaneously.