The protocol gives step-by-step instructions, starting with patient-derived pluripotent stem cells, transitioning them to retinal pigment epithelium (RPE) cells and finally maturing them into RPE tissue. The RPE supports the retina’s light-sensing photoreceptors. The same protocol is being used to generate RPE for an ongoing clinical trial at NIH’s Clinical Center in Bethesda, Maryland, for patients with dry age-related macular degeneration (AMD).
“The protocol begins with induced pluripotent stem (iPS) cells. These are cells modified from patient blood cells that can be turned into nearly any other cell type, including RPE.,” said Ruchi Sharma, Ph.D., staff scientist, NEI Ocular and Stem Cell Translational Unit, and first author of a report on the protocol. "Included are quality control assays for all crucial steps.”
The RPE is crucial to maintaining the health and function of the retina. In dry AMD, RPE function becomes compromised, causing photoceptor degeneration. AMD is one of the most common causes of vision loss in the U.S. But while researchers have discovered a variety of genetic and lifestyle risk factors, no treatment exists for dry AMD.
“Sharing our RPE protocol helps the research community develop human-based research models to study the pathophysiology of AMD and other degenerative retinal diseases,” said Kapil Bharti, Ph.D., who directs the NEI Ocular and Stem Cell Translational Unit and is senior author of the protocol.
To stimulate and facilitate AMD research, the NEI launched the AMD Integrative Biology Initiative, which gives researchers access to patient data and patient-derived cell lines from the NEI-funded Age-related Eye Disease Study 2 (AREDS2)—a major clinical study of AMD. In partnership with the New York Stem Cell Foundation (NYSCF), NEI has developed more than 60 induced-pluripotent stem cell lines from AREDS2 participants. Cell lines are available through the NYSCF. Corresponding patient data, including genetics and retinal imaging results, are available to researchers through the NEI Data Commons.
Sharma R, Bose D, Montford J, Ortolan D, Bharti K. Triphasic developmentally guided protocol to generate retinal pigment epithelium from induced pluripotent stem cells. STAR Protoc. Sep 16 2022;3(3):101582. doi:10.1016/j.xpro.2022.101582
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