Gene therapy to improve muscle function and survival in ALS patients
Gene Therapy Targeting of CNTFRalpha and CLC in Muscle to Treat ALS
This study is testing a new gene therapy that aims to boost certain proteins in the muscles of people with ALS to help protect their motor neurons, with the hope of slowing down the disease and improving their quality of life.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Cincinnati NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project ID | NIH-10634588 on NIH RePORTER |
What this research studies
This research focuses on enhancing the expression of specific proteins in muscle that may protect motor neurons in patients with Amyotrophic Lateral Sclerosis (ALS). By using a gene therapy approach, the study aims to increase levels of ciliary neurotrophic factor receptor α (CNTFRα) and cardiotrophin-like cytokine (CLC) in muscle tissue, which has shown promise in animal models for extending survival and improving motor function. The methodology involves administering a viral vector to deliver these proteins directly to the muscle, potentially offering a new treatment option for ALS patients. If successful, this approach could provide a significant therapeutic benefit by slowing disease progression and improving quality of life.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Amyotrophic Lateral Sclerosis, particularly those who have not yet started advanced treatment.
Not a fit: Patients with advanced stages of ALS or those who have already undergone extensive treatment may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that slows the progression of ALS and improves motor function in patients.
How similar studies have performed: Previous research has shown promising results in animal models using similar gene therapy approaches, indicating potential for success in human applications.
Where this research is happening
Cincinnati, United States
- University of Cincinnati — Cincinnati, United States (Active)
Researchers
- Principal investigator: Maclennan, Alexander John — University of Cincinnati
- Study coordinator: Maclennan, Alexander John
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.