The goal of the Unit on Ocular and Stem Cell Translational Research is to perform translational research on degenerative eye diseases using induced pluripotent stem (iPS) cell technology. We are using this technology to develop in vitro disease models to study patient-specific disease processes, to set up high through drug screens, and to develop cell-based therapy for retinal degenerative disease.
Our translational goals are focused on retinal pigment epithelium (RPE), a monolayer of highly polarized cells located in the back of the eye, whose apical processes inter-digitate with photoreceptor outer segments. RPE performs several functions that are absolutely critical for the health and integrity of photoreceptors. Some of these functions include regulating nutrient and metabolite flow, maintaining ionic homeostasis in the sub-retinal space, regenerating visual pigment, and phagocytizing shed photoreceptor outer segments.
Dysfunctions in the RPE are thought to be the initiating events leading to degenerative eye diseases. Therefore, a better understanding of the disease initiating pathways in RPE will provide a basis for therapeutic interventions. In collaboration with the NEI clinic, we are obtaining skin biopsies from patients with clinically diagnosed degenerative eye diseases. These biopsies are being used to derive iPS cells. RPE cells differentiated from such iPS cells are used to study events that have led to disease initiation and progression.
In collaboration with National Center for Advancing Translational Sciences (NCATS), we have combined the patient-specific iPSC approach with high throughput screening assays performed in 384-well plates to identify novel compounds that could act as potential therapeutic agents. In collaboration with new NIH Center for Regenerative Medicine, we are developing iPSC-derived RPE tissue for cell-based therapy. We have modified the existing stem cell to RPE differentiation protocols to make them more complaint with current Good Manufacturing Practices (cGMP-work).
Our work uses the most cutting edge technologies in the field and aims to translate these technologies to a clinical use.
Last Reviewed: November 2012