University of California receives $13.9M grant from CIRM to advance research into autism and schizophrenia using human stem cell-based models

31 March 2026

Mohammed Mostajo-Radji's team at the University of California Santa Cruz Genomics Institute is to receive a $13.9M grant from the California Institute for Regenerative Medicine to advance preclinical research into autism and schizophrenia.

Both autism and schizophrenia are neurodevelopmental and neuropsychiatric disorders that cause severe impairment in social, educational, occupational and other important areas of functioning. They are uniquely human disorders, with heterogenous, complex etiology and clinical presentations that cannot be faithfully recapitulated in animal models. As a result, the molecular and cellular mechanisms that underpin disease onset and progression remain yet to be fully understood and leveraged to develop treatments that meaningful improve the lives of patients with autism and schizophrenia.

To capture the genetic heterogeneity of these disorders, the multidisciplinary team of scientists will employ human stem cell-based models in two parallel scientific strategies.

The first strategy will focus on well-documented genetic variants for which the link to autism or schizophrenia was already established. By introducing these variants into human stem cell models, directing their growth to brain tissues, and measuring morphological and  electrophysiological properties of resulting brain-like structures, researchers will be able to determine how each genetic variant affects brain development.

“Neuropsychiatric disorders are linked to hundreds of different genetic mutations, and so one of the big questions is how changes in so many different parts of a cell can lead to similar conditions,” Mostajo-Radji said. “This work asks whether these different mutations can lead to similar changes in how brain cells communicate and function as a network. We are aiming to understand how mutations affect brain circuits, and ultimately create better ways to study and treat these conditions.”

Since both conditions involve complex interactions of dozens of variants with unknown effects, the second strategy will employ stem cell-derived brain cells from more than 100 individuals with autism/schizophrenia and neurotypical controls to study associations between specific variants, polygenic risk scores, cellular phenotypes and disease status. To eliminate the variability and artefacts that may arise from heterogenous culturing conditions, the team will pool all donor-derived cells in the same dish.

“By putting everyone’s cells together in the same dish, we improve our ability to detect meaningful biological differences between patients and neurotypical controls,” said Michael F. Wells, a co-investigator on the grant and an assistant professor of human genetics at UCLA. “We also reduce costs dramatically: what might otherwise require tens of thousands of sequencing samples can be done with dozens to hundreds.”

Subsequently, the obtained in vitro models will be used to test the ability of compounds to reverse the cellular phenotypes observed, thereby supporting future clinical trials for both conditions.

For weekly news, trends and insights on human‑based in vitro approaches, explore your access options.

Explore Access