Using viral therapies to treat ALS by targeting harmful proteins
Viral-based Therapeutic Approaches for Reversal of ALS Pathology
This study is looking at new ways to treat ALS by using a special virus to help move a harmful protein back to where it belongs in nerve cells, which could help protect your motor neurons and improve your health.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-10883755 on NIH RePORTER |
What this research studies
This research investigates innovative viral-based therapies aimed at reversing the pathology of amyotrophic lateral sclerosis (ALS), a severe neurodegenerative disease. The approach focuses on relocating a toxic protein, TDP-43, back to the nucleus of nerve cells to mitigate its harmful effects. By utilizing adeno-associated viral vectors, the study aims to deliver therapeutic agents that can effectively reduce the levels of this protein and improve motor neuron health. The research also explores the potential of combining different therapeutic strategies to enhance treatment efficacy.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with ALS who are experiencing motor neuron degeneration.
Not a fit: Patients with ALS who are in advanced stages of the disease or those with other complicating health conditions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new, effective treatments for ALS that significantly improve patient outcomes and quality of life.
How similar studies have performed: Previous research has shown promising results using similar viral vector approaches in ALS models, indicating potential for success in this novel application.
Where this research is happening
Philadelphia, United States
- University of Pennsylvania — Philadelphia, United States (Active)
Researchers
- Principal investigator: Amado, Defne Audrey — University of Pennsylvania
- Study coordinator: Amado, Defne Audrey
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.