Tracking gene therapy effects using advanced MRI technology
Real-Time Tracking of Gene Therapy by Bioactivated MR contrast Probes
['FUNDING_R01'] · NORTHWESTERN UNIVERSITY · NIH-10849633
This study is testing a new MRI technology that helps doctors see how well gene therapy is working in patients with lysosomal storage diseases, so they can better track the treatment's effects on different parts of the body.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | NORTHWESTERN UNIVERSITY (nih funded) |
| Locations | 1 site (Chicago, UNITED STATES) |
| Trial ID | NIH-10849633 on ClinicalTrials.gov |
What this research studies
This research focuses on developing a new magnetic resonance imaging (MRI) technology that allows for real-time tracking of gene therapy effects in patients with lysosomal storage diseases (LSD). By using bioactivated MR contrast probes, the study aims to non-invasively monitor the distribution and activity of adeno-associated virus (AAV) therapies in various organs and the nervous system. This approach addresses the current limitations in assessing treatment efficacy and could lead to improved management of these rare genetic disorders. Patients will be monitored over time to evaluate how well the gene therapy is working.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with lysosomal storage diseases who are undergoing or considering gene therapy.
Not a fit: Patients with conditions unrelated to lysosomal storage diseases or those not receiving gene therapy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a non-invasive method to assess the effectiveness of gene therapies, leading to better treatment outcomes for patients with lysosomal storage diseases.
How similar studies have performed: Other research has shown promising results in using MRI technology for tracking therapeutic effects, but this specific approach is novel and untested.
Where this research is happening
Chicago, UNITED STATES
- NORTHWESTERN UNIVERSITY — Chicago, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MEADE, THOMAS J — NORTHWESTERN UNIVERSITY
- Study coordinator: MEADE, THOMAS J
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.