Understanding neuronopathic lysosomal storage disorders
Systems-Level Approach to Neuronopathic Lysosomal Storage Disorders
This project uses genetic tools and artificial intelligence to find how inherited lysosomal diseases damage brain cells and to point to new treatments for people with neuronopathic lysosomal storage disorders.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project ID | NIH-11173681 on NIH RePORTER |
What this research studies
This research will use mouse models of two different neuronopathic lysosomal storage disorders to map how faulty lysosomes change communication within and between brain cells. Researchers will apply new genetic tools to alter specific lysosomal pathways in different cell types and use AI-driven analysis to find patterns in large, complex datasets. The team aims to identify which lysosomal signals cause neurons and supporting brain cells to fail and which cell types drive disease spread. Those findings could guide development of therapies that target harmful signaling rather than only removing stored material.
Who could benefit from this research
Good fit: People affected by neuronopathic lysosomal storage disorders and their families are the patient group most relevant to this work, though the project is primarily preclinical.
Not a fit: Patients without neuronopathic lysosomal storage disorders or those seeking immediate clinical treatment are unlikely to receive direct benefit from this preclinical research.
Why it matters
Potential benefit: If successful, the work could identify new cellular communication targets that lead to therapies for people with neuronopathic lysosomal storage disorders.
How similar studies have performed: Some preclinical work has shown that changing lysosomal pathways can alter disease in animal models, but using AI to map lysosomal communication across cell types is a newer approach.
Where this research is happening
Saint Louis, United States
- Washington University — Saint Louis, United States (Active)
Researchers
- Principal investigator: Dickson, Patricia I — Washington University
- Study coordinator: Dickson, Patricia I
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.