Investigating the role of a specific genetic mutation in ALS and FTD
Official title: Targeting transcription and translation of the antisense CCCCGG repeat expansion in C9ORF72 ALS/FTD
Funded at University of Chicago · NIH-11071208 · Data updated 2026-11-12
This study is looking into a specific gene change that leads to ALS and FTD, aiming to understand how a certain RNA produced by this gene might harm nerve cells, with the hope of finding new treatments that could help patients like you.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Chicago NIH-funded |
| Lab location | 1 site (Chicago, United States) |
| Project start | January 1, 2025 |
| Project end | December 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $48,974 |
| NIH project number | 1F31NS141619-01 |
| Project ID | NIH-11071208 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with ALS or FTD, particularly those with the C9ORF72 genetic mutation.
✕Less likely to help
Patients without the C9ORF72 mutation or those with other forms of ALS or FTD may not benefit from this research.
What this research studies
This research focuses on understanding the genetic mutation in the C9ORF72 gene that causes amyotrophic lateral sclerosis (ALS) and frontotemporal degeneration (FTD). It aims to explore how the antisense CCCCGG RNA is produced and its potential toxic effects on nerve cells. By identifying the transcription start site of this RNA, the researchers hope to develop new therapeutic strategies targeting this aspect of the disease. Patients may benefit from insights that could lead to innovative treatments for these currently incurable conditions.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic approaches that specifically target the harmful effects of the CCCCGG RNA in ALS and FTD patients.
How similar studies have gone: While there has been significant research on the sense GGGGCC transcript, this investigation into the antisense CCCCGG RNA is relatively novel and has not been extensively tested in clinical settings.
Where this research is happening
-
University of Chicago
Chicago, United States Active
Researchers
- Principal investigator: Antonopoulos, Mira N — University of Chicago
- Study coordinator: Antonopoulos, Mira N
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.