One-time gene therapy for Parkinson's without known genetic causes
Gene Therapy for Idiopathic Parkinson's Disease
A one-time gene treatment using a harmless virus to try to stop disease progression in people with idiopathic Parkinson's disease (no known genetic cause).
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mark Therapeutics, INC. NIH-funded |
| Lab location | 1 site (San Diego, United States) |
| Project ID | NIH-11176818 on NIH RePORTER |
What this research studies
This work is developing a one-time gene therapy that delivers a healthy copy of a protein (PLA2G6L) into dopamine-producing brain cells using an adeno-associated viral (AAV) carrier. In mouse models that mimic age-related idiopathic Parkinson's, a single dose preserved dopamine neurons and slowed motor decline. The company achieved proof-of-concept and now plans preclinical optimization and validation of improved candidate therapies. These steps are intended to prepare the approach for eventual human clinical trials.
Who could benefit from this research
Good fit: Ideal candidates would be people diagnosed with idiopathic Parkinson's disease who do not have known genetic mutations or a family history and who might be eligible for future clinical trials.
Not a fit: People with Parkinson's caused by known genetic mutations, those with medical conditions that make gene therapy unsafe, or anyone seeking immediate treatment (this grant supports preclinical work) may not benefit now.
Why it matters
Potential benefit: If successful, this could stop or greatly slow loss of dopamine neurons and the related movement problems in people with idiopathic Parkinson's.
How similar studies have performed: AAV-based gene therapies have been tested in Parkinson's with mixed results, and targeting PLA2G6L is a novel approach currently supported mainly by mouse preclinical data.
Where this research is happening
San Diego, United States
- Mark Therapeutics, INC. — San Diego, United States (Active)
Researchers
- Principal investigator: Bolotina, Victoria M — Mark Therapeutics, INC.
- Study coordinator: Bolotina, Victoria M
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.