Targeting RNA Splicing Abnormalities in ALS/FTD
Official title: Targeting Dysregulated RNA Splicing in Neurodegenerative Diseases
Funded at Massachusetts General Hospital · NIH-11597831 · Data updated 2026-11-12
A research team aims to understand and correct abnormal RNA splicing in ALS and related brain diseases to help patients in the future.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 1, 2023 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $686,338 |
| NIH project number | 3RM1NS133601-04S1 |
| Project ID | NIH-11597831 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are adults with ALS or related neurodegenerative conditions who may contribute to patient-derived cell studies or be eligible for future translational trials.
✕Less likely to help
Patients without known ALS/FTD or without access to related healthcare resources may not directly benefit from this basic science study.
What this research studies
This project uses patient-derived stem cell models of ALS/FTD to study how mislocalized RNA binding proteins alter RNA splicing across several neuron-related cell types. The team will apply advanced long-read RNA sequencing to map disease-specific splicing changes and identify key mis-spliced genes. By comparing different RNA-binding proteins and cell types, they hope to uncover mechanisms that drive neurodegeneration and test strategies to reverse harmful splicing patterns. The work is designed to build a foundation that could lead to new therapeutic approaches for ALS and FTD in people affected by these conditions.
Why it matters
Potential benefit: If successful, the research could reveal targets to slow or halt neurodegeneration in ALS/FTD and guide future treatments for patients.
How similar studies have gone: Previous work has shown that correcting mis-spliced transcripts can improve neuronal function in model systems, supporting continued exploration in humans.
Where this research is happening
-
Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: Lagier-Tourenne, Clotilde — Massachusetts General Hospital
- Study coordinator: Lagier-Tourenne, Clotilde
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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.