Investigating gene therapy and enzyme inhibitors for treating Krabbe disease
CGT and ACD Inhibitors for SRT Treatment of Krabbe Disease
This study is looking at new ways to help people with Krabbe disease by testing a special gene therapy and other treatments to see if they can improve health and quality of life, especially for those who can’t use the usual treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Illinois at Chicago NIH-funded |
| Lab location | 1 site (Chicago, UNITED STATES) |
| Project ID | NIH-10910053 on NIH RePORTER |
What this research studies
This research focuses on Krabbe disease, a genetic disorder caused by a deficiency in the enzyme galactosylceramidase, leading to toxic accumulation of psychosine in the nervous system. The study aims to explore the effectiveness of adeno-associated viral (AAV) gene therapy and acid ceramidase inhibitors in treating this condition. By utilizing pre-clinical models, the research seeks to improve treatment outcomes for patients, particularly those who are not eligible for current standard treatments like hematopoietic stem cell transplantation. The goal is to enhance survival rates and quality of life for individuals affected by Krabbe disease.
Who could benefit from this research
Good fit: Ideal candidates for this research include infants, children, and adults diagnosed with Krabbe disease, particularly those who are not responding well to current treatments.
Not a fit: Patients with advanced Krabbe disease who are already symptomatic and have progressed beyond the early stages may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a novel treatment option that significantly improves the quality of life and survival rates for patients with Krabbe disease.
How similar studies have performed: Previous studies using AAV gene therapy in animal models have shown promising results, indicating potential for success in human applications.
Where this research is happening
Chicago, UNITED STATES
- University of Illinois at Chicago — Chicago, United States (Active)
Researchers
- Principal investigator: Bongarzone, Ernesto Roque — University of Illinois at Chicago
- Study coordinator: Bongarzone, Ernesto Roque
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.