Finding new treatments for Niemann-Pick Type C disease
Therapeutic Targets for Niemann-Pick Type C Neurodegeneration
This study is looking at Niemann-Pick Type C disease to find new ways to help people with this condition by understanding how a specific gene affects cholesterol in brain cells, since there are no treatments available right now.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project ID | NIH-10908282 on NIH RePORTER |
What this research studies
This research focuses on Niemann-Pick Type C (NPC) disease, a severe genetic disorder that leads to neurodegeneration and early death. The team aims to identify critical targets in the disease's progression that can be used to develop new therapies. By studying the role of the NPC1 gene and its impact on cholesterol trafficking in brain cells, the researchers hope to discover ways to reduce harmful lipid accumulation and improve patient outcomes. This work is essential as there are currently no approved treatments for NPC.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with Niemann-Pick Type C disease, particularly those with mutations in the NPC1 gene.
Not a fit: Patients with other forms of neurodegenerative diseases unrelated to Niemann-Pick Type C may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to the development of effective therapies that modify the course of Niemann-Pick Type C disease.
How similar studies have performed: While there is ongoing research in related areas, this specific approach to targeting NPC disease is novel and has not been extensively tested.
Where this research is happening
Ann Arbor, United States
- University of Michigan at Ann Arbor — Ann Arbor, United States (Active)
Researchers
- Principal investigator: Lieberman, Andrew P — University of Michigan at Ann Arbor
- Study coordinator: Lieberman, Andrew 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.