UCLA Center for Rapid Testing of Genes Linked to Neuropsychiatric Disorders

UCLA High-Throughput Neuropsychiatric Disorder Phenotyping Center (UCLA HT-NPC)

['FUNDING_OTHER'] · UNIVERSITY OF CALIFORNIA LOS ANGELES · NIH-11377079

The team will use human stem cells to turn off hundreds of genes linked to autism and other neuropsychiatric conditions to find how those changes affect developing brain cells.

Quick facts

Phase['FUNDING_OTHER']
Study typeNih_funding
SexAll
SponsorUNIVERSITY OF CALIFORNIA LOS ANGELES (nih funded)
Locations1 site (LOS ANGELES, UNITED STATES)
Trial IDNIH-11377079 on ClinicalTrials.gov

What this research studies

Researchers will create 'null' (turned-off) versions of about 250 genes implicated in neuropsychiatric and neurodevelopmental disorders using human embryonic and induced pluripotent stem cells. They will test whether edited cells survive and can become neurons, then measure molecular and cellular changes using RNA sequencing and other quantitative phenotyping. Promising gene knockouts will be turned into clonal cell lines for deeper study, combining rapid screening with detailed follow-up to map how specific genes influence brain-cell development and function.

Who could benefit from this research

Good fit: People with autism spectrum disorder or other neurodevelopmental/neuropsychiatric conditions—especially those with known genetic variants—would be the most relevant candidates to donate samples or join related follow-up studies.

Not a fit: Those without neurodevelopmental or neuropsychiatric conditions, or anyone seeking immediate clinical treatment, are unlikely to benefit directly from this laboratory-focused project.

Why it matters

Potential benefit: If successful, this work could reveal which genes disrupt brain development and point to new targets for diagnosis, monitoring, or future therapies.

How similar studies have performed: Prior lab studies using stem cells and gene editing have clarified effects of some risk genes, but applying high-throughput knockouts and systematic phenotyping across hundreds of genes is a newer and more ambitious approach.

Where this research is happening

LOS ANGELES, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.