Stem cell derived retinal pigment epithelial cells as a cell-replacement therapy for age-related macular degeneration
The advanced dry form of age-macular degeneration is one of the most important causes of blindness in the Western countries. The disease of unknown etiology causes the loss of the retinal pigment epithelial cell (RPE) layer, which leads to subsequent degeneration of essential retinal structures (e.g. photoreceptors) causing severe vision impairment. Our work aims to:
i) Derive RPE cells from human embryonic stem cells (hESC) in the most pure and efficient manner.
ii) Assess the behavior and integration / functionality of the derived cells in a relevant preclinical model of disease (eg rabbit eye) entailing surgical and imaging methods as closely as possible to those applied in humans.
iii) Characterize and modulate the immunological properties of the derived hESC-RPE to create a minimally immunogenic cell source via CRISPR-Cas9 technology.
We hope that all these advances in regenerative medicine can serve as a safe, efficient and minimally invasive replacement therapy to treat patients suffering from age-related macular degeneration, as well as a platform for other similar stem cell-based therapies intending to reach the clinics.