iPSC: A potential candidate for the regeneration of retinal pigment epithelium in retinal degenerative diseases

Age-related macular degeneration (AMD), retinitis pigmentosa (RP) and Stargardt’s disease (SD) are the leading causes of permanent blindness in developed countries. Thus far, gene therapy and growth factor supplement strategies cannot regenerate the retinal pigment epithelial cells (RPE) lost during the development of these diseases. Cell therapy replacement strategies such the use of foetal tissues and embryonic stem cells (ESCs) are still subject to ethical concerns and tumorigenic-related responses associated with the use of foetal material.

The central aim of this project is to identify the best method of differentiation from patient cells into RPE and PR cells. The development of such technologies will enable the start of a phase I clinical trial in AMD, RP and SD patients.

Expected results: The generation of the different cellular products will enable the development of a phase I clinical trial in AMD, RP and SD patients.


(1) Embryonic Stem Cell Therapy – From bench to bed. By Laura E. Sperling
(2) Rodman & Renshaw Global Investment Conference September 2012