Improving enzyme function for Parkinson's disease and dementia treatment
Official title: Validation of Small Molecule Enhancers of Mutant Glucocerebrosidase 1 Activity
Funded at Johns Hopkins University · NIH-10988959 · Data updated 2026-11-12
This study is looking for new ways to help a key enzyme that doesn't work properly in people with Parkinson's disease and Dementia with Lewy Bodies, by finding small molecules that can help it get to where it needs to be in the brain, which could lead to better treatments for these conditions.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | July 1, 2024 |
| Project end | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $163,750 |
| NIH project number | 1R03NS135450-01A1 |
| Project ID | NIH-10988959 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Parkinson's disease or Dementia with Lewy Bodies, particularly those with the L444P mutation in the GBA1 gene.
✕Less likely to help
Patients without genetic mutations related to glucocerebrosidase 1 or those with other forms of dementia unrelated to this enzyme may not benefit from this research.
What this research studies
This research focuses on enhancing the activity of a specific enzyme, glucocerebrosidase 1, which is crucial for patients with Parkinson's disease and Dementia with Lewy Bodies. The study aims to develop small molecule chaperones that can help misfolded versions of this enzyme reach their proper location in the cell, potentially improving its function. By using high-throughput screening methods, researchers will identify compounds that can cross the blood-brain barrier and assist in the treatment of these neurodegenerative conditions. Patients may benefit from new therapeutic options that address the underlying enzyme deficiency.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that improve the quality of life for patients with Parkinson's disease and Dementia with Lewy Bodies.
How similar studies have gone: Previous research has shown promise in using molecular chaperones for similar enzyme deficiencies, indicating a potential for success in this novel approach.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Ko, Hanseok — Johns Hopkins University
- Study coordinator: Ko, Hanseok
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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.