Limbal stem cells for treatment of corneal wounds in Epidermolysis Bullosa
| Investigator(s) | Prof Jakub Tolar, University of Minnesota |
| Funding | US $250,000 (£165,000) Cure EB (formerly the Sohana Research Fund under DEBRA UK) |
| Start date | January 2014 |
| End date | August 2015 |
| Research area | Gene editing, Cell therapy |
| Aim | Quality of life change |
| EB sub-type | Dystrophic EB |
| Status | Complete |
Lay summary
The cornea, the transparent outer surface of the eye which covers the iris and pupil, is important in bending light that enters the eye into a precise point, enabling clear vision. The cornea is fragile and frequently damaged in Recessive Dystrophic Epidermolysis Bullosa (RDEB) by trauma as minor as opening the eyes after sleep. This can lead to irreversible damage, excruciating pain, and blindness.
Induced pluripotent stem (iPS) cells are a type of stem cell generated from other adult cells. Like other stem cells, they have the ability to become another type of cell in the body. In this project, human skin and eye cells were reprogrammed into iPS cells, which were then turned into limbal stem cells (corneal stem cells) with functioning collagen VII.
Scientific Summary
Hematopoietic cell transplant cannot reach all parts of the body. The cornea, the main structure for refraction of light entering the eye, is a prime example. The cornea is frequently damaged in RDEB, and those affected are left with one of the most overwhelmingly irreversible ravages of this disease in terms of both pain and blindness.
In this proof-of-concept study, researchers developed a platform technology whereby human corneal epithelial cells (limbal stem cells) were derived from a limited source of differentiated cells via reprogramming to pluripotency and subsequent differentiation – now significantly amplified cell numbers – human corneal epithelial cells.
This technology has the potential to support both autologous and allogeneic grafts.
