Inherited and acquired eye diseases that culminate in the degeneration of photoreceptors and retinal pigment epithelium (RPE) are a significant cause of visual morbidity. The expansion and targeted differentiation of human stem and progenitor cells in vitro provide an essential source of biological material for modeling retinal development and potential cell-based treatments for these debilitating diseases. The aims of our laboratory are to 1) investigate cellular and molecular events that occur during retinogenesis and 2) provide cells for use in rescue or replacement therapies for retinal degenerative diseases.

To meet these goals, we utilize a variety of cell types.  Retinal and neural progenitors are amenable to viral transformation and demonstrate the capacity to efficiently deliver neuroprotective factors directly to the retina. Human embryonic stem cells (hESCs) are used to delineate the genetic “checkpoints” necessary to produce a particular retinal cell type and serve as a model system for studying human retinal development. Lastly, in collaboration with Dr. James Thomson, our laboratory has initiated a study designed to direct induced pluripotent stem cells (iPS) towards a retinal lineage in a manner similar to hESCs, which may allow for the creation of cell-based models for human retinal degenerative diseases.  By understanding the behavior of these cell types in vitro and in vivo, we hope to optimize strategies to delay or reverse the effects of inherited and acquired eye diseases such as retinitis pigmentosa and macular degeneration.