Helping cells stop harmful protein misfolding in the ER
Mechanisms regulating ER Proteostasis
['FUNDING_R01'] · TUFTS UNIVERSITY BOSTON · NIH-11249249
Looking for ways cells prevent bad protein folding to help people with cystic fibrosis and some neurodegenerative diseases.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | TUFTS UNIVERSITY BOSTON (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11249249 on ClinicalTrials.gov |
What this research studies
This project looks inside cells' protein-making compartment, the endoplasmic reticulum (ER), to find molecules that prevent protein misfolding before it happens. The team studies how cells control protein production at the ribosome and how misfolded proteins are tagged for destruction prior to entering the ER. Researchers use cellular and biochemical experiments to identify the proteins and pathways that drive this pre-emptive quality control. The goal is to reveal targets that could be used to stop toxic protein buildup in multiple diseases.
Who could benefit from this research
Good fit: People with diseases linked to ER protein misfolding—such as cystic fibrosis or certain neurodegenerative disorders—would be the most relevant candidates to follow this research or contribute samples.
Not a fit: Patients whose conditions are unrelated to ER protein folding, and anyone seeking an immediate clinical treatment, are unlikely to receive direct benefit from this laboratory-based work.
Why it matters
Potential benefit: If successful, this could lead to new treatments that boost the cell's natural quality-control system to reduce harmful protein buildup in conditions like cystic fibrosis and neurodegeneration.
How similar studies have performed: Past research has characterized the unfolded protein response and other proteostasis pathways and has produced some successful therapies, but targeting pre-emptive ER quality control is a newer approach with limited prior clinical testing.
Where this research is happening
BOSTON, UNITED STATES
- TUFTS UNIVERSITY BOSTON — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: RAMAN, MALAVIKA — TUFTS UNIVERSITY BOSTON
- Study coordinator: RAMAN, MALAVIKA
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.