A new gene therapy to stop protein misfolding in multiple system atrophy.
A Novel Gene Therapy Approach to Prevent Alpha-synuclein Misfolding in Multiple System Atrophy
This study is exploring a new gene therapy that uses CRISPR technology to create a special version of a protein linked to multiple system atrophy (MSA), with the goal of stopping the disease from getting worse.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Colorado State University NIH-funded |
| Lab location | 1 site (Fort Collins, United States) |
| Project ID | NIH-10922836 on NIH RePORTER |
What this research studies
This research investigates a novel gene therapy approach aimed at preventing the misfolding of the α-synuclein protein, which is responsible for the progression of multiple system atrophy (MSA). The team will utilize CRISPR technology to create a modified version of the α-synuclein protein that cannot misfold, potentially halting the disease's progression. By introducing specific changes to the protein's genetic sequence, the researchers hope to disrupt the harmful self-templating process that leads to neurodegeneration. This innovative approach has not yet been tested in therapeutic settings for MSA, making it a pioneering effort in the field.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with multiple system atrophy who are experiencing symptoms related to the disease.
Not a fit: Patients with other neurodegenerative disorders that do not involve α-synuclein misfolding may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that slows or stops the progression of multiple system atrophy.
How similar studies have performed: While gene therapy approaches for neurodegenerative diseases are being explored, this specific method targeting α-synuclein misfolding in MSA is novel and has not been previously tested.
Where this research is happening
Fort Collins, United States
- Colorado State University — Fort Collins, United States (Active)
Researchers
- Principal investigator: Woerman, Amanda L. — Colorado State University
- Study coordinator: Woerman, Amanda L.
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.