Boosting CHD2 levels to help CHD2-related autism and epilepsy

Bidirectional control of Chd2 haploinsufficiency

NIH-funded research University of California-Irvine · NIH-11263721

Researchers are working to increase CHD2 protein in brain cells to see whether that could help people with CHD2-related autism, intellectual disability, or epilepsy.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of California-Irvine NIH-funded
Lab location1 site (Irvine, United States)
Project IDNIH-11263721 on NIH RePORTER

What this research studies

You would be learning about research that changes CHD2 levels in lab-grown human neurons and in mice engineered to carry CHD2 mutations. Scientists will use genetic tools, drugs, and brain recordings to raise or lower CHD2 during development or in adulthood and then measure effects on gene activity, nerve cell function, and behavior. The team aims to find whether increasing CHD2 can reverse disease-linked changes and point toward a drug-based therapy. Successful findings would support moving toward clinical trials for people with CHD2 haploinsufficiency.

Who could benefit from this research

Good fit: Ideal candidates would be individuals with a confirmed CHD2 loss-of-function mutation or diagnosis of CHD2 haploinsufficiency, often presenting with autism, intellectual disability, and/or epilepsy.

Not a fit: People whose autism or epilepsy is not caused by CHD2 mutations are unlikely to benefit directly from this work.

Why it matters

Potential benefit: If successful, this work could lead to a drug approach that raises CHD2 and improves seizures, learning, or autism-related differences in people with CHD2 haploinsufficiency.

How similar studies have performed: Prior mouse studies from this group and others have linked CHD2 loss to brain and behavioral problems, but using drugs to raise CHD2 as a therapy is a novel, early-stage approach.

Where this research is happening

Irvine, 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.