Creating targeted therapies for ALS and FTD using specific RNA techniques
Official title: Development of Mutant Specific Antisense Oligonucleotides for TARDBP and KIF5A as a Therapeutic for ALS/FTD
Funded at Univ of Massachusetts Med Sch Worcester · NIH-10976216 · Data updated 2026-11-12
This study is working on new treatments for ALS and Frontotemporal Dementia by targeting the specific gene changes that cause these conditions, aiming to help patients by blocking the harmful effects of those genes while keeping the healthy ones working.
Quick facts
| Grant type | R21 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 | June 1, 2024 |
| Project end | May 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $460,625 |
| NIH project number | 1R21NS139270-01 |
| Project ID | NIH-10976216 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with ALS or FTD who have specific genetic mutations related to these diseases.
✕Less likely to help
Patients with ALS or FTD who do not have the targeted genetic mutations may not benefit from this research.
What this research studies
This research focuses on developing new gene-based therapies for Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) by targeting specific mutations in genes associated with these conditions. The approach involves creating antisense oligonucleotides that can selectively inhibit the harmful effects of mutant genes while preserving the function of normal genes. By utilizing advanced RNA interference techniques, the researchers aim to provide a more effective treatment option for patients with these genetic disorders. The project will explore the mechanisms of gene mutations and how they contribute to disease progression, ultimately leading to innovative therapeutic strategies.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for ALS and FTD, potentially improving patient outcomes and quality of life.
How similar studies have gone: Previous research has shown promise in using RNA interference techniques for gene-specific therapies, indicating potential success for this novel approach.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Landers, John E — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Landers, John E
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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.