We propose preclinical studies necessary to gain FDA approval for a Phase 1 clinical trial using human embryonic stem cell (ESC) derived retinal cells (including photoreceptors) to treat retinal degeneration in end stage Retinitis Pigmentosa (RP). Currently there are no treatments available to slow or prevent the relentless course of these diseases. It is our goal to replace the lost retinal cells in individuals with these diseases using retinal cells derived from human ES cells.
RP is inherited retinal diseases that lead to visual impairment and blindness in over in many California residents and a total of 100,000 individuals in the US. While there are many diverse causes of these diseases, they have as their common outcome the degeneration of the retinal cells. Gene therapy has shown promising result only in a few patients with a single gene mutation, while majority of patients are still left no treatment. Crucially, progressive retinal degeneration leads to untreatable blindness; and once the retinal cells have degenerated, there are no means for their replacement. Individual with end stage disease can only hope that some means for replacing the lost cells can be found. Our study has the potential to replace lost retinal cells in individuals with these diseases and restore vision.