Gene therapy approach for treating Menkes disease
Official title: VIRAL GENE THERAPY FOR MENKES DISEASE
Funded at Battelle Centers/pub Hlth Res & Evaluatn · NIH-11215799 · Data updated 2026-11-12
This study is testing a new gene therapy for Menkes disease, which could help people by fixing the genetic issue that causes problems with copper in the body, and it's currently being tried out in animals to make sure it's safe and effective before moving on to humans.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Battelle Centers/pub Hlth Res & Evaluatn NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project start | September 30, 2024 |
| Project end | September 29, 2027 |
| Funding year | 2024 |
| Award (this year) | $91,138 |
| NIH project number | 75N95022D00021-0-759502400003-1 |
| Project ID | NIH-11215799 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Menkes disease or those who are carriers of the genetic mutation associated with the condition.
✕Less likely to help
Patients with other unrelated genetic disorders or those who do not have Menkes disease may not receive any benefit from this research.
What this research studies
This research focuses on developing a viral gene therapy to treat Menkes disease, a genetic disorder that affects copper metabolism. The study involves pre-clinical animal models to assess the safety, efficacy, and potential toxic effects of the therapy before it can be tested in humans. By using advanced genetic intervention techniques, the research aims to correct the underlying genetic defect responsible for the disease. Patients may benefit from this innovative approach if it proves successful in restoring normal copper levels in the body.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients with Menkes disease, potentially improving their quality of life and health outcomes.
How similar studies have gone: While gene therapy is a relatively novel approach for Menkes disease, similar strategies have shown promise in treating other genetic disorders, indicating potential for success.
Where this research is happening
-
Battelle Centers/pub Hlth Res & Evaluatn
Columbus, United States Active
Researchers
- Principal investigator: Burnaugh, Amanda — Battelle Centers/pub Hlth Res & Evaluatn
- Study coordinator: Burnaugh, Amanda
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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.