Skin cell therapy for recessive dystrophic epidermolysis bullosa
Skin-targeted Cell Therapy for Recessive Dystrophic Epidermolysis Bullosa
This project tries to correct the faulty COL7A1 gene in skin cells to reduce blistering for people with recessive dystrophic epidermolysis bullosa (RDEB).
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Minnesota NIH-funded |
| Lab location | 1 site (Minneapolis, United States) |
| Project ID | NIH-11130138 on NIH RePORTER |
What this research studies
You would learn about treatments that aim to fix the COL7A1 gene in skin cells using advanced gene-editing tools like prime editors and engineered integrases. The team plans to test both ex vivo approaches (repairing cells in the lab and putting them back on the skin) and in‑body delivery using viral vectors such as non‑integrating HSV. The focus is on making precise, site-specific corrections that keep normal gene control and lower the risk of harmful DNA insertions. Safety, durability, and cancer risk concerns will be closely monitored in preclinical work before broader patient use.
Who could benefit from this research
Good fit: People with confirmed recessive dystrophic epidermolysis bullosa caused by COL7A1 mutations would be the ideal candidates.
Not a fit: People with other types of epidermolysis bullosa not caused by COL7A1 mutations or those unable to undergo skin sampling or gene therapy procedures may not benefit.
Why it matters
Potential benefit: If successful, this could restore collagen VII in the skin and substantially reduce blistering, scarring, and wound complications for people with RDEB.
How similar studies have performed: Other viral and cell therapy approaches for RDEB have shown early promise in clinical reports, but the prime-assisted integrase and locus-specific editing methods described here are relatively new.
Where this research is happening
Minneapolis, United States
- University of Minnesota — Minneapolis, United States (Active)
Researchers
- Principal investigator: Tolar, Jakub — University of Minnesota
- Study coordinator: Tolar, Jakub
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.