A new gene therapy to stop protein misfolding in multiple system atrophy.
Official title: A Novel Gene Therapy Approach to Prevent Alpha-synuclein Misfolding in Multiple System Atrophy
Funded at Colorado State University · NIH-10922836 · Data updated 2026-11-12
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 start | September 15, 2023 |
| Project end | August 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $192,144 |
| NIH project number | 5R21NS127002-02 |
| Project ID | NIH-10922836 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with multiple system atrophy who are experiencing symptoms related to the disease.
✕Less likely to help
Patients with other neurodegenerative disorders that do not involve α-synuclein misfolding may not benefit from this research.
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.
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 gone: 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
-
Colorado State University
Fort Collins, United States Active
Researchers
- Principal investigator: Woerman, Amanda L. — Colorado State University
- Study coordinator: Woerman, Amanda L.
Related research
Other NIH-funded projects on the same topics.
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Integrated MRI biomarkers to diagnose and monitor multiple system atrophy
This project will help doctors use MRI markers to better diagnose multiple system atrophy and track how the disease progresses.
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Clinical Trial Readiness for Multiple System Atrophy
A multi-center study to help people with multiple system atrophy by creating better clinical and biological measures to use in future drug trials.
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Single-cell brain profiling to map MSA cell changes
Researchers will map gene activity in individual brain cells from people who had multiple system atrophy to understand how the disease affects cells in key brain regions.
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Investigating how inflammation caused by alpha-synuclein affects multiple system atrophy
This study is looking at how inflammation affects multiple system atrophy (MSA) and aims to understand how the immune system might play a role in the disease, with the hope of finding new ways to help people with MSA.
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Understanding brain inflammation in multiple system atrophy
This research explores how a specific immune signal, interferon-gamma, contributes to brain damage in multiple system atrophy.
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Single-cell gene map of brains from people with multiple system atrophy
This project reads gene activity in individual brain cells from people with multiple system atrophy to find cell-specific changes linked to the disease.
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.