Investigating new treatments for autism using genetic insights and stem cells
Official title: Epigenetics-Based Autism Treatment with Animal Models and Human Stem Cells
Funded at State University of New York at Buffalo · NIH-11012797 · Data updated 2026-11-12
This study is looking for new medications to help improve social skills and reduce repetitive behaviors in people with autism by investigating a gene called Shank3, using mice and human brain cells to find out what treatments might work best.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | State University of New York at Buffalo NIH-funded |
| Lab location | 1 site (Amherst, United States) |
| Project start | February 15, 2023 |
| Project end | January 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $556,576 |
| NIH project number | 5R01NS127728-03 |
| Project ID | NIH-11012797 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with autism spectrum disorder, particularly those with genetic factors related to the Shank3 gene.
✕Less likely to help
Patients without autism spectrum disorder or those whose symptoms are not linked to genetic factors related to the Shank3 gene may not benefit from this research.
What this research studies
This research focuses on developing new pharmacological treatments for autism by exploring the role of the Shank3 gene, which is linked to social deficits and repetitive behaviors in autism. The team will utilize mouse models lacking the Shank3 gene and human stem cell-derived neurons to test potential drug interventions. By examining epigenetic changes and the effects of specific agents on gene expression, the research aims to identify effective therapies that could improve symptoms associated with autism. The approach combines behavioral assessments, biochemical analyses, and advanced genomic techniques to validate findings.
Why it matters
Potential benefit: If successful, this research could lead to innovative treatments that significantly improve social interactions and reduce repetitive behaviors in individuals with autism.
How similar studies have gone: Previous research has shown promise in using genetic and epigenetic approaches to address autism-related symptoms, indicating that this methodology has potential for success.
Where this research is happening
-
State University of New York at Buffalo
Amherst, United States Active
Researchers
- Principal investigator: Yan, Zhen — State University of New York at Buffalo
- Study coordinator: Yan, Zhen
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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.