Induced pluripotent stem cell-derived autologous cell therapy for age-related macular degeneration

Abstract

Photoreceptor cell death associated with advanced age-related macular degeneration (AMD) is triggered by degeneration of retinal pigment epithelium (RPE). Replacement of the atrophied RPE cell layer has previously shown potential to preserve visual acuity in autograft surgeries. However, these procedures are risky and complicated. RPE cells derived from patient-specific induced pluripotent stem (iPS) cells can be reproducibly manufactured as a tissue on a surgically compatible substrate and transplanted back into the patient's eye potentially with no risk of immune rejection. Using a developmentally guided differentiation protocol we have manufactured autologous RPE tissue from AMD patients on a biodegradable scaffold. In vitro\, this tissue demonstrates morphological, molecular, and functional attributes that are similar to the native tissue. We have confirmed safety and efficacy of this tissue in vivo in an RPE injury pig model. Our results provide a potential treatment for AMD using autologous iPS cells.