Gene Profiling Technique Accelerates Stem Cell Therapies for Retinal Diseases

(Source: National Eye Institute, National Institutes of Health)
Researchers at the National Institutes of Health have developed a technique that will streamline the production of stem-cell derived tissues. The technique will help the researchers in their efforts to use patients’ skin cells to create new retinal tissue, a therapy that has been recently applied to a human subject in Japan. It will also help the scientists search for drugs for personalized treatments.
As described in a recent issue of Stem Cells Translational Medicine, the identity of retinal pigment epithelium (RPE) made from stem cells is verified by using microscopy to ensure the tissue looks like retinal pigment epithelium and physiological assays to ensure the tissue behaves like RPE. A technique called quantitative RT-PCR is also used to measure the expression of genes that indicate ongoing cell development and function. But this permits only the simultaneous measurement of a few genes per sample.
To measure more genes simultaneously, a new method has been developed by Dr. Kapil Bharti, Ph.D. (Stadtman Investigator in the Unit on Ocular and Stem Cell Translational Research at the National Eye Institute) and Marc Ferrer, Ph.D. (National Center for Advancing Translational Sciences). With the new method, they were able to cut costs in half and increase the output of tissue.
Dr. Bharti and colleagues at NEI are now collaborating with the New York Stem Cell Foundation to produce stem cell lines from patients with hereditary eye diseases. These cell lines will be available to the entire scientific community through a central repository, from which Dr. Bharti will generate RPE to study age-related macular degeneration.
More information about Dr. Bharti’s research, including a video, is available on the NIH Intramural Research Program’s website.
Reference: Ferrer M, Corneo B, Davis J, et al. A Multiplex High-Throughput Gene Expression Assay to Simultaneously Detect Disease and Functional Markers in Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium. Stem Cells Translational Medicine. May 2014.