Exploring how a cellular process affects TDP-43 in neurodegenerative diseases.
Investigating the Roles of the Deubiquitylation Pathway in TDP-43 Pathology in ALS/FTD
['FUNDING_R21'] · UNIV OF MASSACHUSETTS MED SCH WORCESTER · NIH-11187363
This study is looking at how a specific process in our cells affects a protein called TDP-43, which is connected to diseases like ALS and frontotemporal dementia, to find new ways to help treat these conditions.
Quick facts
| Phase | ['FUNDING_R21'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIV OF MASSACHUSETTS MED SCH WORCESTER (nih funded) |
| Locations | 1 site (WORCESTER, UNITED STATES) |
| Trial ID | NIH-11187363 on ClinicalTrials.gov |
What this research studies
This research investigates the role of the deubiquitylation pathway in the pathology of TDP-43, a protein linked to neurodegenerative diseases like ALS and frontotemporal dementia. Using advanced techniques such as mouse models and CRISPR-edited human neurons, the study aims to understand how this pathway influences the localization and aggregation of TDP-43. By uncovering these mechanisms, the research seeks to identify potential new therapeutic targets for treating these challenging conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with ALS, frontotemporal dementia, or Alzheimer's disease, particularly those with TDP-43 pathology.
Not a fit: Patients with neurodegenerative diseases not associated with TDP-43 pathology may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatment strategies for patients suffering from ALS, FTD, and related neurodegenerative diseases.
How similar studies have performed: While the specific approach of targeting the deubiquitylation pathway in TDP-43 pathology is relatively novel, similar research has shown promise in understanding neurodegenerative diseases.
Where this research is happening
WORCESTER, UNITED STATES
- UNIV OF MASSACHUSETTS MED SCH WORCESTER — WORCESTER, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: GAO, FEN-BIAO — UNIV OF MASSACHUSETTS MED SCH WORCESTER
- Study coordinator: GAO, FEN-BIAO
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: Alzheimer disease dementia, Alzheimer syndrome