The ability to generate induced Pluripotent Stem Cell (iPSC) from skin and also peripheral blood of individuals allows the exciting possibility of making patient - and disease-specific models to study disease mechanisms and test new therapies. This is particularly important when appropriate animal models are not practical or available. Recently, advances have been made that allowed differentiation of human iPSC into photoreceptors and their nurse cells, the retinal-pigmented epithelium (RPE). Part of the Scheie Eye Institute, CAROT has the capability to bank cells from patients seen in our clinics with inherited retinal degenerations, and establish a valuable biobank which can be used to identify new disease genes as well as to create iPSC that can be used to model a large set of orphan diseases. At CAROT, we have used patient-derived iPSC to provide proof of concept data for gene augmentation therapy for Choroideremia, an X-linked recessive retinal disease caused by mutation and loss of function of CHM which encodes Rab Escort Protein1. Thus, the ability of CAROT to create a biobank of cells from patients with inherited retinal degenerations and to generate patient-specific models would open the door to a personalized approach to understanding and treating inherited retinal degenerations.
The Induced Pluripotent Stem Cell Core is directed by Jeannette Bennicelli.
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