Multi-Target RNA Therapies for ALS and Alzheimer's Disease
Official title: Multi-Targeting Oligonucleotide Therapeutics for Familial and Sporadic Amyotrophic Lateral Sclerosis
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11329236 · Data updated 2026-11-12
A patient-friendly look at a project that aims to develop RNA-based medicines to slow or modify ALS and Alzheimer’s-related diseases by targeting multiple genes.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | May 15, 2019 |
| Project end | May 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $665,737 |
| NIH project number | 5R01NS111990-08 |
| Project ID | NIH-11329236 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with ALS or Alzheimer’s-related dementia who are candidates for RNA-targeted therapies or related clinical trials.
✕Less likely to help
Many patients may not benefit in the near term if the therapies are not safe or effective in humans or if biomarkers do not translate.
What this research studies
This grant focuses on creating RNA-targeted medicines, like ASOs and siRNAs, to silence harmful genes involved in ALS and Alzheimer’s disease. Researchers plan to address safety and effectiveness in the nervous system, and explore combinations of gene targets to improve outcomes. The work involves preclinical and early translational steps that could lead to future therapies for people affected by these conditions. If successful, the approach could provide a new way to reduce disease-causing protein production in the brain and spinal cord.
Why it matters
Potential benefit: Could lead to new RNA-based treatments that slow progression or modify the course of ALS and Alzheimer’s-related conditions.
How similar studies have gone: RNA-targeted medicines have shown promise, with some approvals for other diseases and ongoing trials for neurodegenerative disorders.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Watts, Jonathan K — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Watts, Jonathan K
Related research
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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.