Gene Therapy for AADC Deficiency
A Safety and Efficacy Study of AAV2-hAADC for AADC Deficiency
['FUNDING_R01'] · OHIO STATE UNIVERSITY · NIH-11197578
This research explores a gene therapy to help children with AADC deficiency by delivering a missing enzyme to their brain.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | OHIO STATE UNIVERSITY (nih funded) |
| Locations | 1 site (Columbus, UNITED STATES) |
| Trial ID | NIH-11197578 on ClinicalTrials.gov |
What this research studies
AADC deficiency is a serious genetic condition that causes severe movement problems and developmental delays. This gene therapy aims to deliver a working copy of the AADC enzyme directly to the brain using a special virus. The goal is to correct the underlying cause of the disease and improve symptoms. Previous treatments have shown promising results, with patients experiencing fewer severe episodes and gaining better motor control.
Who could benefit from this research
Good fit: Ideal candidates are children diagnosed with AADC deficiency who may be experiencing severe neurological symptoms.
Not a fit: Patients without AADC deficiency or those with different underlying genetic conditions would not receive benefit from this specific gene therapy.
Why it matters
Potential benefit: If successful, this gene therapy could offer a disease-modifying treatment that significantly improves the quality of life for children with AADC deficiency.
How similar studies have performed: Previous treatments in a small group of patients have shown positive results, including improved motor skills and reduced severe symptoms.
Where this research is happening
Columbus, UNITED STATES
- OHIO STATE UNIVERSITY — Columbus, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BANKIEWICZ, KRYSTOF S — OHIO STATE UNIVERSITY
- Study coordinator: BANKIEWICZ, KRYSTOF S
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.