RSSH donors to help Fe-S cluster formation in Friedreich's ataxia cells
Official title: Mechanistic Investigations into Hydropersulfide (RSSH) Donor-Mediated Rescue of Deficient Iron-Sulfur Cluster Biosynthesis in Friedreich's Ataxia Fibroblasts
Funded at Johns Hopkins University · NIH-11342528 · Data updated 2026-11-12
A scientist is testing whether hydropersulfide donors can restore iron-sulfur cluster formation in skin cells from people with Friedreich's ataxia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | June 1, 2026 |
| Project end | February 28, 2030 |
| Funding year | 2026 |
| Award (this year) | $325,665 |
| NIH project number | 1R01GM164444-01 |
| Project ID | NIH-11342528 on NIH RePORTER |
Who could benefit from this research
✓Could help
FA patient-derived skin fibroblasts (with FXN deficiency) and control fibroblasts.
✕Less likely to help
Cells from non-Friedreich's ataxia individuals with normal FXN function may not show rescue.
What this research studies
This project will use cells from Friedreich's ataxia patients to see if hydropersulfide donors can fix the faulty iron-sulfur cluster production that impairs mitochondria. Researchers will compare treated patient cells to healthy control cells to understand the protective effects on Fe-S biosynthesis. The study will analyze cluster assembly steps, mitochondrial health, and cellular stress responses to determine potential rescue mechanisms. If successful, it could point toward strategies to relieve cellular problems caused by FXN deficiency.
Why it matters
Potential benefit: If successful, this work could uncover a new approach to protect or restore mitochondrial function in Friedreich's ataxia.
How similar studies have gone: Previous research has begun detailing Fe-S cluster assembly and the role of sulfur transfer; this study extends that by testing RSSH donors for functional rescue.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Toscano, John P — Johns Hopkins University
- Study coordinator: Toscano, John 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.