UCLA researchers receive $13.9 million CIRM grant to identify drug targets for autism and schizophrenia using human stem cell models
Published in Diseases!.
A team of researchers led by UCLA’s Dr. Daniel Geschwind has received a $13.9 million grant from the to use human stem cell-based models to uncover the molecular and cellular mechanisms underlying autism and schizophrenia and to identify drug targets for both conditions.
These conditions affect tens of millions of people in the U.S., yet no drugs exist that target their underlying biology — largely because researchers don’t yet know precisely how brain cells from people with these conditions differ, at the molecular level, from those of neurotypical individuals, leaving nothing concrete to screen drugs against.
“Right now, there’s virtually no roadmap for developing treatments for these conditions,” said Geschwind, a distinguished professor of human genetics, neurology and psychiatry at the and member of the . “This work is focused on solving that problem by studying many variants in parallel, which will provide an initial roadmap for our lab, for other researchers, and for the pharmaceutical companies that could eventually take these findings into the clinic.”
Both conditions are thought to have strong genetic components that emerge as early as prenatal brain development, but those early molecular differences have never been systematically characterized across a representative patient population — leaving the drug development pipeline effectively stalled before it starts.
To address this gap, Geschwind and his multidisciplinary team will pursue two parallel scientific strategies, each targeting a different dimension of genetic risk. The first focuses on specific, well-documented genetic variants already known to raise the risk of autism or schizophrenia.
Using CRISPR gene editing, the team will introduce 36 autism-linked variants and 32 schizophrenia-linked variants — several of which are shared between the two conditions — into human stem cells, then coax those cells to grow into miniature, brain-like structures.
https://www.uclahealth.org/news/release/ucla-researchers-receive-139-million-cirm-grant-identify