Investigating how autism risk genes affect brain cell function

Convergent cellular and network mechanisms of ASD risk genes

['FUNDING_CAREER'] · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · NIH-10994629

This study is looking at how certain genes linked to autism affect special brain cells that help with thinking and social skills, and it hopes to find new ways to help people with autism through gene therapy.

Quick facts

Phase['FUNDING_CAREER']
Study typeNih_funding
SexAll
SponsorUNIVERSITY OF CALIFORNIA, SAN FRANCISCO (nih funded)
Locations1 site (SAN FRANCISCO, UNITED STATES)
Trial IDNIH-10994629 on ClinicalTrials.gov

What this research studies

This research focuses on understanding how various genes associated with autism spectrum disorder (ASD) impact the function of specific brain cells known as layer 5 pyramidal neurons in the prefrontal cortex. By examining these neurons, the research aims to uncover how disruptions in their excitability and synaptic integration contribute to the social and cognitive challenges faced by individuals with ASD. The study will also explore the potential of gene therapy to address these disruptions caused by genetic factors. Patients may benefit from insights that could lead to new therapeutic strategies for ASD.

Who could benefit from this research

Good fit: Ideal candidates for this research are adults diagnosed with autism spectrum disorder who may have genetic factors contributing to their condition.

Not a fit: Patients without a diagnosis of autism spectrum disorder or those whose condition is not linked to genetic factors may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to innovative treatments that improve brain function and social skills in individuals with autism.

How similar studies have performed: Previous research has shown promise in understanding the genetic underpinnings of autism, making this approach both relevant and potentially impactful.

Where this research is happening

SAN FRANCISCO, 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.