Investigating gene-targeted therapies for autism and related disorders
Official title: Exploring Feasibility of Gene-Targeted Therapeutics for Epigenetic Neurodevelopmental Disorders
Funded at Washington University · NIH-11095893 · Data updated 2026-11-12
This study is looking at new ways to help people with autism and related developmental disorders by fixing certain gene problems after birth, using special mouse models to see if these treatments could really make a difference.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project start | June 1, 2024 |
| Project end | May 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $155,500 |
| NIH project number | 5R21NS137088-02 |
| Project ID | NIH-11095893 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with autism or neurodevelopmental disorders caused by specific genetic mutations.
✕Less likely to help
Patients with neurodevelopmental disorders not linked to genetic mutations or those with severe developmental delays may not benefit from this research.
What this research studies
This research explores the potential of gene-targeted therapies to treat autism and neurodevelopmental disorders (ASD/NDDs) by restoring gene activity after birth. It focuses on understanding how specific genetic mutations, particularly those affecting 'epigenetic writer' enzymes, contribute to these conditions. By developing specialized mouse models, the research aims to test the feasibility of correcting genetic defects that lead to ASD/NDDs, which could pave the way for innovative treatments. The approach is based on recent findings that suggest postnatal interventions may be effective in ameliorating symptoms associated with these disorders.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic options for individuals with autism and related neurodevelopmental disorders.
How similar studies have gone: While the approach of targeting genetic therapies for ASD/NDDs is relatively novel, preliminary studies in animal models have shown promising results.
Where this research is happening
-
Washington University
Saint Louis, United States Active
Researchers
- Principal investigator: Gabel, Harrison W — Washington University
- Study coordinator: Gabel, Harrison W
Related research
Other NIH-funded projects on the same topics.
-
Intergenerational factors in neurodevelopmental disorders
This project looks at how experiences before and during pregnancy across generations may influence the risk of neurodevelopmental conditions like ADHD, autism, and related disorders in grandchildren.
-
Understanding brain signals in autism to identify biologically distinct subtypes
This study uses EEG to link how the brain processes sensory information with behavior in young children with autism and related concerns.
-
Clinical Translational Research Center for Neurodevelopmental Disorders
A funded center supports research on neurodevelopmental disorders like Angelman syndrome and autism to better understand causes and improve treatments for children.
-
Synaptic nanostructure in neurodevelopmental disorders
This project aims to map the tiny architecture of brain synapses in native cells to understand how connections form and change in neurodevelopmental disorders.
-
Maternal acetaminophen use in pregnancy and ADHD risk in children
A large study will follow thousands of mother-child pairs to see if taking acetaminophen during pregnancy relates to ADHD in kids.
-
GDF11’s role in brain development and autism-related disorders
A new NIH grant tests whether adjusting a growth factor called GDF11 can improve brain function and behaviors in models related to autism and other neurodevelopmental disorders.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.