Understanding how an immune pathway causes brain damage in Niemann-Pick Disease
Mechanism of STING-mediated Neuropathology in Niemann-Pick Disease
['FUNDING_R01'] · UT SOUTHWESTERN MEDICAL CENTER · NIH-11127732
This project explores how a specific immune pathway contributes to brain damage in Niemann-Pick disease, a condition sometimes called 'Childhood Alzheimer's'.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UT SOUTHWESTERN MEDICAL CENTER (nih funded) |
| Locations | 1 site (DALLAS, UNITED STATES) |
| Trial ID | NIH-11127732 on ClinicalTrials.gov |
What this research studies
Niemann-Pick disease type C (NPC) is a serious brain condition with no approved treatments, often showing symptoms similar to Alzheimer's disease. We've found that an immune pathway called STING becomes overactive in NPC and seems to cause brain damage. This work aims to uncover exactly how STING causes this damage, looking at both how it gets activated inside cells and how it affects brain tissue. We will also test existing and new medicines that block the STING pathway in patient-derived cells and animal models to see if they can protect the brain.
Who could benefit from this research
Good fit: This foundational research focuses on understanding disease mechanisms and testing potential treatments in models, so direct patient participation is not currently part of this specific grant.
Not a fit: Patients seeking immediate clinical trial enrollment for Niemann-Pick disease would not find direct opportunities within this basic science project.
Why it matters
Potential benefit: If successful, this work could lead to new treatments that target the STING pathway, offering hope for patients with Niemann-Pick disease and potentially other neurodegenerative conditions.
How similar studies have performed: While the STING pathway has been linked to other neurodegenerative diseases, its specific role and therapeutic targeting in Niemann-Pick disease are novel and currently being explored.
Where this research is happening
DALLAS, UNITED STATES
- UT SOUTHWESTERN MEDICAL CENTER — DALLAS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: YAN, NAN — UT SOUTHWESTERN MEDICAL CENTER
- Study coordinator: YAN, NAN
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