Understanding how a protein called Ataxin-2 affects ALS and FTD
Role of Ataxin-2 polyglutamine expansion on TDP-43 transport and post-transcriptional RNA regulation in neurons
['FUNDING_R01'] · YALE UNIVERSITY · NIH-11103203
This project aims to understand how changes in a protein called Ataxin-2 contribute to nerve cell damage in people with ALS and frontotemporal dementia.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | YALE UNIVERSITY (nih funded) |
| Locations | 1 site (NEW HAVEN, UNITED STATES) |
| Trial ID | NIH-11103203 on ClinicalTrials.gov |
What this research studies
Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are serious brain diseases that currently have limited treatments. We know that a protein called TDP-43 forms harmful clumps in almost all ALS patients and many FTD patients. This project focuses on how another protein, Ataxin-2, interacts with TDP-43 and affects how nerve cells handle RNA, which is crucial for cell function. By understanding these interactions, we hope to uncover the root causes of nerve cell damage in these conditions.
Who could benefit from this research
Good fit: This foundational research is not directly recruiting patients but focuses on understanding the disease mechanisms relevant to individuals with ALS and FTD.
Not a fit: Patients who do not have ALS or FTD, or those whose conditions are not linked to TDP-43 or Ataxin-2 changes, may not directly benefit from this specific line of research.
Why it matters
Potential benefit: If successful, this work could reveal new targets for developing treatments that slow or stop the progression of ALS and FTD.
How similar studies have performed: While the specific mechanisms of Ataxin-2 and TDP-43 interaction are still being explored, other studies have highlighted the importance of RNA-binding proteins in neurodegenerative diseases.
Where this research is happening
NEW HAVEN, UNITED STATES
- YALE UNIVERSITY — NEW HAVEN, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: GOPAL, PALLAVI P. — YALE UNIVERSITY
- Study coordinator: GOPAL, PALLAVI P.
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