Understanding genetic changes in ALS to find new treatment targets
Official title: Delineating the functional impact of recurrent repeat expansions in ALS using integrative multiomic analysis
Funded at Columbia University Health Sciences · NIH-10776994 · Data updated 2026-11-12
This study is looking at the genetic changes in people with ALS to find out how certain DNA patterns might affect the disease and help us discover new ways to treat it.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | September 20, 2023 |
| Project end | September 19, 2025 |
| Funding year | 2023 |
| Award (this year) | $323,817 |
| NIH project number | 1R03OD036491-01 |
| Project ID | NIH-10776994 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with ALS and those with a family history of the disease.
✕Less likely to help
Patients with other neurodegenerative disorders that do not involve genetic repeat expansions may not benefit from this research.
What this research studies
This research investigates the genetic alterations associated with Amyotrophic Lateral Sclerosis (ALS), a serious neurodegenerative disease. By analyzing whole genome sequencing data from ALS patients and healthy individuals, the study aims to identify recurrent DNA repeat expansions that may play a role in the disease's progression. The researchers will integrate various types of biological data, including gene expression and chromatin accessibility, to understand how these genetic changes affect motor neuron health. This comprehensive approach could lead to new insights into potential therapeutic targets for ALS.
Why it matters
Potential benefit: If successful, this research could lead to the development of new treatments that target the underlying genetic causes of ALS.
How similar studies have gone: Previous research has shown promise in identifying genetic markers in neurodegenerative diseases, suggesting that this approach could yield valuable insights.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Gursoy, Gamze — Columbia University Health Sciences
- Study coordinator: Gursoy, Gamze
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.