Use of human pluripotent stem cells to study and treatretinopathies

• 作者: Karim Ben M’Barek Florian Regent Christelle Monville
• 来源期刊: 世界干细胞杂志：英文版（电子版）
• 学科: 医学
• 关键词: DRUG SCREENING Human PLURIPOTENT STEMCELLS Disease modeling RETINITIS pigmentosa
• 年，卷（期）: 2015,（3）
• 页码范围: 596-604
• 页数: 9页
• 分类号: R

摘要

Human cell types affected by retinal diseases （such asage-related macular degeneration or retinitis pimentosa）are limited in cell number and of reduced accessibility. As aconsequence, their isolation for in vitro studies of diseasemechanisms or for drug screening efforts is fastidious.Human pluripotent stem cells （hPSCs）, either of embryonicorigin or through reprogramming of adult somatic cells,represent a new promising way to generate models ofhuman retinopathies, explore the physiopathologicalmechanisms and develop novel therapeutic strategies.Disease-specific human embryonic stem cells were thefirst source of material to be used to study certain diseasestates. The recent demonstration that human somaticcells, such as fibroblasts or blood cells, can be geneticallyconverted to induce pluripotent stem cells together withthe continuous improvement of methods to differentiatethese cells into disease-affected cellular subtypes opensnew perspectives to model and understand a largenumber of human pathologies, including retinopathies.This review focuses on the added value of hPSCs for thedisease modeling of human retinopathies and the study oftheir molecular pathological mechanisms. We also discussthe recent use of these cells for establishing the validationstudies for therapeutic intervention and for the screeningof large compound libraries to identify candidate drugs.