Understanding how certain genetic changes cause nerve cell damage in ALS and frontotemporal dementia
Mechanisms of chimeric DPR production and toxicity in c9ALS/FTD
['FUNDING_R01'] · EMORY UNIVERSITY · NIH-11128738
This study is looking into how a specific gene mutation causes harmful proteins to form in people with ALS and Frontotemporal Dementia, with the hope of finding new treatment options that could help improve their lives.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | EMORY UNIVERSITY (nih funded) |
| Locations | 1 site (ATLANTA, UNITED STATES) |
| Trial ID | NIH-11128738 on ClinicalTrials.gov |
What this research studies
This research investigates the genetic mechanisms behind the production of toxic proteins in patients with Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD). It focuses on a specific genetic mutation in the C9orf72 gene that leads to the formation of harmful dipeptide repeat proteins. By exploring how these proteins are produced and their effects on nerve cells, the research aims to uncover potential targets for new treatments. Patients may benefit from insights that could lead to innovative therapies for these debilitating conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with ALS or FTD, particularly those with the C9orf72 genetic mutation.
Not a fit: Patients with ALS or FTD who do not have the C9orf72 mutation may not benefit directly from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatment strategies that slow down or halt the progression of ALS and FTD.
How similar studies have performed: Previous research has shown promising results in understanding genetic mechanisms in similar neurodegenerative disorders, indicating potential for success in this area.
Where this research is happening
ATLANTA, UNITED STATES
- EMORY UNIVERSITY — ATLANTA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MCEACHIN, ZACHARY THOMAS — EMORY UNIVERSITY
- Study coordinator: MCEACHIN, ZACHARY THOMAS
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 below.