Gene therapy aimed at treating idiopathic Parkinson's Disease

Who could benefit from this research

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.

Why it matters

Where this research is happening

• Mark Therapeutics, INC.
  San Diego, United States Active

Researchers

• Principal investigator: Bolotina, Victoria M — Mark Therapeutics, INC.
• Study coordinator: Bolotina, Victoria M

Related research

Other NIH-funded projects on the same topics.

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About this research

1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.