NIH discovery brings stem cell therapy for eye disease closer to the clinic

5 January 2018
National Institutes of Health

Scientists at the National Eye Institute (NEI), part of the National Institutes of Health, report that tiny tube-like protrusions called primary cilia on cells of the retinal pigment epithelium (RPE) – a layer of cells in the back of the eye – are essential for the survival of the retina’s light-sensing photoreceptors. The discovery has advanced effore to make stem cell-derived RPE for transplantation into patients with geographic atrophy, otherwise known as dry age-related macular degeneration (AMD), a leading cause of blindness in the U.S The study appears in the January 2 Cell Reports.

In a healthy eye, RPE cells nourish and support photoreceptors, the cells that convert light into electrical signals that travel to the brain via the optic nerve. RPE cells form a layer just behind the photoreceptors. In geographic atrophy, RPE cells die, which causes photoreceptors to degenerate, leading to vision loss.

Kapil Bharti PhD, Stadtman investigator at the NEI, and colleagues are hoping to halt and reverse the progression of geographic atrophy by replacing diseased RPE with lab-made RPE. The approach involves using a patient’s blood cells to generate induced-pluripotent stem cells (iPSCs), cells capable of becoming any type of cell in the body. iPSCs are grown in the laboratory and then coaxed into becoming RPE for surgical implantation.

For more information on the discovery, Click here