Understanding how lysosomal disorders affect brain function
Official title: Systems-Level Approach to Neuronopathic Lysosomal Storage Disorders
Funded at Washington University · NIH-10924008 · Data updated 2026-11-12
This study is looking at how problems with cell parts called lysosomes affect the progression of certain serious brain diseases, and it's for people with neuronopathic lysosomal storage disorders; researchers hope to find new ways to treat these conditions by understanding how lysosomes communicate in cells.
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 start | September 15, 2023 |
| Project end | August 31, 2028 |
| Funding year | 2024 |
| Award (this year) | $1,558,704 |
| NIH project number | 5RM1NS132962-02 |
| Project ID | NIH-10924008 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with neuronopathic lysosomal storage disorders, particularly those with genetic mutations affecting lysosomal function.
✕Less likely to help
Patients with non-neuronopathic lysosomal storage disorders or those without genetic involvement in lysosomal function may not benefit from this research.
What this research studies
This research investigates neuronopathic lysosomal storage disorders (LSDs), which are severe neurodegenerative diseases caused by genetic defects in lysosomes. The study aims to explore how changes in lysosomal communication within cells, rather than just the accumulation of toxic materials, contribute to disease progression. Using advanced genetic tools and artificial intelligence, researchers will analyze lysosomal behavior in mouse models to identify potential new therapeutic targets. This approach could lead to a better understanding of the disease mechanisms and pave the way for innovative treatments.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that improve outcomes for patients with lysosomal storage disorders.
How similar studies have gone: While the approach of using AI to analyze lysosomal communication is innovative, previous research has shown promise in understanding lysosomal disorders, indicating potential for success.
Where this research is happening
-
Washington University
Saint Louis, United States Active
Researchers
- Principal investigator: Dickson, Patricia I — Washington University
- Study coordinator: Dickson, Patricia I
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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.