Fixing misplaced proteins in cell membranes
Quality control of mislocalized membrane proteins
['FUNDING_R01'] · HARVARD MEDICAL SCHOOL · NIH-11301902
This project aims to understand how cells find and remove membrane proteins that end up in the wrong place, which could help people with neurodegenerative diseases like Alzheimer's and ALS.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | HARVARD MEDICAL SCHOOL (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11301902 on ClinicalTrials.gov |
What this research studies
Researchers study basic cell processes that keep membrane proteins in the right organelles using simple, single-spanning protein models and lab systems that let them follow protein handling step by step. They focus on a transporter called ATP13A1 that can pull mislocalized mitochondrial membrane proteins out of the wrong membrane. The team uses molecular and cell biology tools to map the quality-control steps that fail in disease. The lab work is meant to reveal mechanisms that could later guide treatments for Alzheimer’s, ALS, and related disorders.
Who could benefit from this research
Good fit: People with Alzheimer's disease, ALS, or related neurodegenerative conditions — or those willing to donate tissue or genetic samples — are the most relevant candidates for future participation.
Not a fit: Patients seeking immediate treatment or those without neurodegenerative conditions are unlikely to receive direct benefit from this laboratory-focused research.
Why it matters
Potential benefit: If successful, this work could point to new drug targets or therapies to prevent organelle dysfunction that contributes to Alzheimer’s, ALS, and other neurodegenerative diseases.
How similar studies have performed: Previous laboratory studies have identified ATP13A1's role in removing mislocalized proteins, so this work builds on emerging mechanistic findings but has not yet produced clinical therapies.
Where this research is happening
BOSTON, UNITED STATES
- HARVARD MEDICAL SCHOOL — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SHAO, SICHEN — HARVARD MEDICAL SCHOOL
- Study coordinator: SHAO, SICHEN
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, Alzheimer's Disease, Amyotrophic Lateral Sclerosis Motor Neuron Disease