Investigating the role of specific proteins in kidney disease related to Tuberous Sclerosis Complex.
Official title: Role of TFEB and TFE3 in Tuberous Sclerosis Complex (TSC) Kidney Disease
Funded at Brigham and Women's Hospital · NIH-11118807 · Data updated 2026-11-12
This study is looking at how certain proteins affect kidney problems in people with Tuberous Sclerosis Complex, with the hope of finding better treatments that could help improve kidney health and reduce the need for long-term medication.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 1, 2022 |
| Project end | April 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $384,263 |
| NIH project number | 5R01DK133354-04 |
| Project ID | NIH-11118807 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with Tuberous Sclerosis Complex, particularly those experiencing kidney-related complications.
✕Less likely to help
Patients without Tuberous Sclerosis Complex or those not experiencing kidney issues may not benefit from this research.
What this research studies
This research focuses on understanding how the proteins TFEB and TFE3 contribute to kidney disease in patients with Tuberous Sclerosis Complex (TSC). By studying the effects of these proteins on kidney tumors and other related conditions, the research aims to identify new therapeutic strategies that could improve patient outcomes. The approach includes analyzing cellular mechanisms and utilizing animal models to observe the impact of these proteins on tumor growth and kidney function. The ultimate goal is to find more effective treatments that could reduce the need for lifelong therapy.
Why it matters
Potential benefit: If successful, this research could lead to novel therapies that effectively eliminate kidney tumors associated with Tuberous Sclerosis Complex.
How similar studies have gone: Previous research has shown promising results in targeting similar pathways in other conditions, suggesting potential for success in this novel approach.
Where this research is happening
-
Brigham and Women's Hospital
Boston, United States Active
Researchers
- Principal investigator: Henske, Elizabeth P — Brigham and Women's Hospital
- Study coordinator: Henske, Elizabeth P
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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.