Use of induced pluripotent stem cell models to probe the pathogenesis of Choroideremia and to develop a potential treatment

Choroideremia (CHM) is a rare monogenic, X-linked recessive inherited retinal degeneration resulting from mutations in the Rab Escort Protein-1 (REP1) encoding CHM gene. The primary retinal cell type leading to CHM is unknown. In this study, we explored the utility of induced pluripotent stem cell-d...

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Bibliographic Details
Main Authors: Thu T. Duong, Vidyullatha Vasireddy, Pavitra Ramachandran, Pamela S. Herrera, Lanfranco Leo, Carrie Merkel, Jean Bennett, Jason A. Mills
Format: Article
Language:English
Published: Elsevier 2018-03-01
Series:Stem Cell Research
Online Access:http://www.sciencedirect.com/science/article/pii/S1873506118300151