CMCB Life Sciences Seminar: Dr. Kapil Bharti, National Eye Institute Bethesda, USA

Host: Marius Ader (CRTD)

Title: “Translating RPE Biology into Disease Treatments Using Stem Cells.”

Abstract: I have more than 15 years of experience in eye development and differentiation of retinal pigment epithelium (RPE). Recently, my lab has capitalized on induced pluripotent stem (iPS) cell technology to develop therapies for degenerative eye diseases. Based on my past experience with RPE development, we optimized a robust protocol to differentiate RPE from human iPS cells. This protocol is highly efficient and reproducible in generating functional RPE cells. Combining this developmental biology approach with tissue engineering, we have grown iPSC-RPE monolayers on a biodegradable scaffold and developed methods to functionally authenticate these monolayers. The resulting iPSC-RPE tissue resembles native tissue in its physiological properties. This work has formed the basis for a phase 1/IIa clinical trial testing the RPE-scaffold as a therapeutic implant in advanced macular degeneration. In parallel, my lab also developed methods and assays to perform large-scale drug screen using iPSC-RPE grown in 384-well plates. These high throughput screens are being used to develop potential therapies for degenerative eye diseases. With our extensive experience in iPSC-RPE, we have derived dozens of iPS lines, which we have extensively characterized and genotyped.