Understanding Protein Changes in Nerve Diseases
Polyalanine Tails: A Novel Type of Protein Modification Implicated in Neurodegeneration
['FUNDING_R01'] · JACKSON LABORATORY · NIH-11093330
This project aims to uncover how certain protein changes contribute to nerve diseases like ALS, helping us find new ways to help patients.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | JACKSON LABORATORY (nih funded) |
| Locations | 1 site (BAR HARBOR, UNITED STATES) |
| Trial ID | NIH-11093330 on ClinicalTrials.gov |
What this research studies
Neurodegenerative diseases, such as ALS, are challenging conditions with limited treatment options, and we don't fully understand what causes them. This project focuses on a process called Ribosome-associated Quality Control (RQC), which helps clear out faulty proteins in our cells. We know that problems with RQC, specifically with a protein called NEMF, can lead to nerve damage in mice and have been found in patients with similar conditions. By learning more about how NEMF dysfunction causes nerve cells to break down, we hope to identify new targets for future therapies.
Who could benefit from this research
Good fit: This foundational research is most relevant to individuals living with or at risk for neurodegenerative conditions like Amyotrophic Lateral Sclerosis (ALS).
Not a fit: Patients seeking immediate new treatments will not directly benefit from this basic science project, as it focuses on understanding disease mechanisms.
Why it matters
Potential benefit: If successful, this work could reveal fundamental causes of neurodegenerative diseases, paving the way for the development of new treatments.
How similar studies have performed: Previous work by the researchers has already linked RQC dysfunction to neurodegeneration in mice and identified similar genetic mutations in human patients, suggesting a promising direction.
Where this research is happening
BAR HARBOR, UNITED STATES
- JACKSON LABORATORY — BAR HARBOR, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BURGESS, ROBERT W. — JACKSON LABORATORY
- Study coordinator: BURGESS, ROBERT W.
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