Investigating how TDP-43 protein is regulated in ALS and frontotemporal dementia
RNA-based regulation of TDP-43 nuclear export in ALS/FTD
['FUNDING_R01'] · JOHNS HOPKINS UNIVERSITY · NIH-10866549
This study is looking at a protein called TDP-43 to understand how it behaves in conditions like ALS and frontotemporal dementia, with the hope of finding new ways to help treat these diseases.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | JOHNS HOPKINS UNIVERSITY (nih funded) |
| Locations | 1 site (BALTIMORE, UNITED STATES) |
| Trial ID | NIH-10866549 on ClinicalTrials.gov |
What this research studies
This research focuses on understanding the role of TDP-43, a protein that is crucial for RNA processing, in the context of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The study aims to uncover the mechanisms behind the mislocalization of TDP-43 from the nucleus to the cytoplasm, which is linked to neurodegeneration in these diseases. By exploring the pathways involved in TDP-43 nuclear export, the research seeks to identify potential therapeutic targets that could help restore normal protein function. Patients may benefit from insights gained that could lead to new treatment strategies for ALS and FTD.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with amyotrophic lateral sclerosis or frontotemporal dementia.
Not a fit: Patients with other neurodegenerative diseases not related to TDP-43 mislocalization may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic approaches for treating ALS and FTD by targeting the mechanisms of TDP-43 mislocalization.
How similar studies have performed: While the specific mechanisms of TDP-43 nuclear export are still being explored, related research has shown promise in understanding protein mislocalization in neurodegenerative diseases.
Where this research is happening
BALTIMORE, UNITED STATES
- JOHNS HOPKINS UNIVERSITY — BALTIMORE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: HAYES, LINDSEY RENAE — JOHNS HOPKINS UNIVERSITY
- Study coordinator: HAYES, LINDSEY RENAE
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.
Conditions: Amyotrophic Lateral Sclerosis Motor Neuron Disease