Investigating gene-targeted therapies for autism and related disorders
Exploring Feasibility of Gene-Targeted Therapeutics for Epigenetic Neurodevelopmental Disorders
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 ID | NIH-11095893 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with autism or neurodevelopmental disorders caused by specific genetic mutations.
Not a fit: Patients with neurodevelopmental disorders not linked to genetic mutations or those with severe developmental delays may not benefit from this research.
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 performed: 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
Saint Louis, United States
- Washington University — Saint Louis, United States (Active)
Researchers
- Principal investigator: Gabel, Harrison W — Washington University
- Study coordinator: Gabel, Harrison W
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 below.