Gene therapy approach for treating Menkes disease
VIRAL GENE THERAPY FOR MENKES DISEASE
['FUNDING_OTHER'] · BATTELLE CENTERS/PUB HLTH RES & EVALUATN · NIH-11215799
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
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BATTELLE CENTERS/PUB HLTH RES & EVALUATN (nih funded) |
| Locations | 1 site (COLUMBUS, UNITED STATES) |
| Trial ID | NIH-11215799 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Menkes disease or those who are carriers of the genetic mutation associated with the condition.
Not a fit: Patients with other unrelated genetic disorders or those who do not have Menkes disease may not receive any benefit from this research.
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 performed: 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
COLUMBUS, UNITED STATES
- 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
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.
Conditions: Disease, Disorder, Kinky Hair Disease, Kinky Hair Syndrome, Menkes Kinky Hair Syndrome