Gene therapy aimed at treating idiopathic Parkinson's Disease
Gene Therapy for Idiopathic Parkinson's Disease
This study is testing a new gene therapy for people with idiopathic Parkinson's Disease, which is a common form of the condition that doesn't run in families, to see if it can help improve brain function and slow down symptoms.
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-10919327 on NIH RePORTER |
What this research studies
This research focuses on developing a novel gene therapy specifically for patients with idiopathic Parkinson's Disease (idPD), which affects around 80% of Parkinson's patients who do not have genetic mutations or a family history of the disease. The approach involves using an Adeno-Associated Viral (AAV) vector to deliver a functional protein that can restore critical cellular functions in dopamine-producing neurons, potentially halting disease progression. The therapy has shown promising results in preclinical models, demonstrating safety and efficacy in slowing motor dysfunction. The research aims to optimize and validate this therapy through rigorous preclinical studies.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with idiopathic Parkinson's Disease who do not have genetic mutations or a family history of the condition.
Not a fit: Patients with Parkinson's Disease caused by known genetic mutations or those with a strong family history of the disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option that modifies the progression of idiopathic Parkinson's Disease.
How similar studies have performed: This approach is novel and has shown initial success in preclinical models, but further validation in human trials is needed.
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.