Understanding Genetic Autism Using Lab-Grown Brain Models

Human Isogenic Organoid Models of Genetic Forms of Autism to Identify Convergent and Divergent Pathomechanisms in Autism

NIH-funded research University of California, San Diego · NIH-11082287

This work uses special lab-grown brain models, created from patient cells, to better understand how different genetic forms of autism develop.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of California, San Diego NIH-funded
Lab location1 site (La Jolla, United States)
Project IDNIH-11082287 on NIH RePORTER

What this research studies

Autism spectrum disorder (ASD) is complex, and finding common causes across its many forms is important for new treatments. This project creates unique 3D brain models, called organoids, from stem cells of patients with specific genetic types of autism. These models help us look closely at how cells communicate and function, especially focusing on how proteins are made, which might be disrupted in autism. By studying these models, we hope to find shared problems in brain development that lead to autism, even in different genetic forms. This approach aims to uncover new ways to diagnose and treat autism.

Who could benefit from this research

Good fit: This foundational research does not directly involve patient participation, but it focuses on understanding genetic forms of autism that affect children, particularly those aged 0-11 years.

Not a fit: Patients whose autism is not linked to the specific genetic forms being modeled in this laboratory setting may not directly benefit from this particular line of research.

Why it matters

Potential benefit: If successful, this work could lead to new ways to diagnose autism and develop more effective treatments by identifying common underlying causes.

How similar studies have performed: While the use of iPSC-derived organoids is an established technique, this project creates the largest panel of isogenic iPSC lines for syndromic ASD and introduces a novel method (ribo-STAMP) for single-cell translational profiling, making its specific approach highly innovative.

Where this research is happening

La Jolla, 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.
Conditions Autistic Disorder
Last reviewed 2026-06-13 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.