Dual-AAV gene replacement approach for SCN2A-related neurodevelopmental disorders
Official title: Dual-AAV gene replacement for SCN2A disorders
Funded at Purdue University · NIH-11594149 · Data updated 2026-11-12
A gene therapy approach using two AAV vectors to restore SCN2A function in children with SCN2A-related neurodevelopmental disorders.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Purdue University NIH-funded |
| Lab location | 1 site (West Lafayette, United States) |
| Project start | August 4, 2026 |
| Project end | July 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $545,644 |
| NIH project number | 1R56NS144233-01A1 |
| Project ID | NIH-11594149 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children with SCN2A-related neurodevelopmental disorders caused by loss-of-function variants.
✕Less likely to help
Patients without SCN2A-related conditions or with non-deficient variants may not benefit from this approach.
What this research studies
This project aims to test a dual-AAV gene replacement strategy to restore SCN2A gene activity in brain cells, using mouse models and human-derived cells. By splitting the SCN2A gene across two AAV vectors, researchers plan to overcome size limits and reconstitute a working sodium channel to improve neural signaling. The work will involve validating delivery, expression, and functional rescue of disease-related phenotypes in models that mimic early childhood onset conditions. Progress could pave the way for a targeted, genetic treatment for affected children.
Why it matters
Potential benefit: If successful, this therapy could restore healthy neuronal function for children with SCN2A-related disorders and potentially reduce developmental and seizure-related symptoms.
How similar studies have gone: Prior work shows that partial gene restoration can reverse disease phenotypes in models; dual-AAV strategies are a promising but still early approach.
Where this research is happening
-
Purdue University
West Lafayette, United States Active
Researchers
- Principal investigator: Yang, Yang — Purdue University
- Study coordinator: Yang, Yang
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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.