ABCB5 stem cells to restore the cornea in limbal stem cell deficiency
ABCB5-positive stem cells for LSCD therapy
This project plans to use ABCB5-positive corneal stem cells to help people with limbal stem cell deficiency regrow their corneal surface and improve vision.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11099688 on NIH RePORTER |
What this research studies
I have limbal stem cell deficiency when the cornea cannot renew itself and vision can be lost; this project focuses on a specific corneal stem cell called ABCB5+ that can rebuild the surface. Researchers can isolate ABCB5+ cells using a targeted antibody, expand them in the lab, and transplant them onto the eye. Earlier work led to clinical use of donor ABCB5+ cells but those required immune-suppressing drugs. This grant will test three new approaches intended to treat people with both eyes affected, including ways to use a patient’s own cells or reduce rejection.
Who could benefit from this research
Good fit: Ideal candidates are people with bilateral limbal stem cell deficiency causing corneal epithelial failure and vision loss.
Not a fit: Patients whose vision loss is due to other ocular diseases, those with active eye infection, or those unable to undergo surgical procedures may not benefit from this approach.
Why it matters
Potential benefit: If successful, this could restore a clear corneal surface and improve vision for people with bilateral LSCD while reducing or eliminating the need for long-term immunosuppression.
How similar studies have performed: Prior clinical work transplanted donor ABCB5+ limbal stem cells (ClinicalTrials.gov NCT03549299) showing feasibility, but autologous, non-immunosuppressed approaches are less established.
Where this research is happening
Boston, United States
- Boston Children's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Frank, Markus H. — Boston Children's Hospital
- Study coordinator: Frank, Markus H.
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.