The University of California San Francisco said Aug. 27 it has received a $46 million grant to broaden its research into the molecular roots of autism spectrum disorder.
Aligning Research to Impact Autism awarded the grant to UCSF’s Quantitative Biosciences Institute, according to a university news release. The funds will advance research published Aug. 27 in Science.
The study “produced the largest molecular interaction map of autism risk genes to date, revealing how hundreds of genetic mutations converge on a surprisingly small number of shared protein complexes,” according to UCSF. “The findings point toward a new generation of precision medicines for autism and other complex disorders.”
Researchers analyzed 100 high-confidence autism risk genes and mapped more than 1,800 protein interactions — 87% of them not previously reported — drawing on 54 patient-derived mutations. They found that genetically distinct forms of autism often converge on the same disrupted protein complexes, suggesting a single therapy could target multiple genetic subtypes at once.
The grant will fund the Quantitative Biosciences Institute’s role in ARIA’s Protein-Protein Interactions Hub, one of six research hubs in the funder’s network, as the institute works to move from molecular mapping to mechanism-based small-molecule drugs. Small molecules could offer advantages in brain delivery, tolerability and manufacturing over gene-targeting approaches such as antisense oligonucleotides or CRISPR, according to UCSF.
“It accelerates our ability to move from molecular insight to therapeutic impact for autism and ultimately for many other diseases,” said Nevan Krogan, PhD, who directs QBI and is leading the institute’s work on the project. ARIA, which backs focused research hubs across the U.S., U.K. and Canada, has centered its mission on expanding therapeutic options for people with profound autism.
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