Targeting a gene linked to a common inherited brain disorder using a new delivery method
Official title: Neuronal Silencing of ATXN3 Using Peripherally Administered Antibody/ASO Conjugates That Penetrate the Blood-Brain Barrier
Funded at University of Michigan at Ann Arbor · NIH-10834266 · Data updated 2026-11-12
This study is testing a new treatment for people with SCA3, a genetic condition that affects movement and coordination, by using a special combination of antibodies and molecules to help block the harmful gene and hopefully slow down the disease's progression.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project start | May 1, 2023 |
| Project end | April 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $195,000 |
| NIH project number | 5R21NS132018-02 |
| Project ID | NIH-10834266 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with SCA3 or those who are genetically predisposed to this condition.
✕Less likely to help
Patients with other forms of ataxia or neurodegenerative disorders unrelated to the ATXN3 gene may not benefit from this research.
What this research studies
This research focuses on a genetic condition known as SCA3, or Machado–Joseph disease, which leads to coordination and movement difficulties. The study aims to suppress the harmful gene responsible for this condition, ATXN3, using a novel approach that combines antibodies with anti-sense oligonucleotides (ASOs). This method allows for intravenous administration, enabling the treatment to cross the blood-brain barrier effectively and target specific neurons in the brain. By addressing the limitations of current therapies, this research seeks to slow or halt the progression of neurodegeneration associated with SCA3.
Why it matters
Potential benefit: If successful, this research could provide a new, less invasive treatment option for patients with SCA3, potentially improving their quality of life.
How similar studies have gone: While the approach of using ASOs has shown promise in other neurodegenerative conditions, this specific method of targeting ATXN3 is relatively novel and untested.
Where this research is happening
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University of Michigan at Ann Arbor
Ann Arbor, United States Active
Researchers
- Principal investigator: Tessier, Peter M — University of Michigan at Ann Arbor
- Study coordinator: Tessier, Peter M
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.