Protective Effects of Human iPS-Derived Retinal Pigment Epithelium Cell Transplantation in the Retinal Dystrophic Rat 英文参考文献.docVIP

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Protective Effects of Human iPS-Derived Retinal Pigment Epithelium Cell Transplantation in the Retinal Dystrophic Rat 英文参考文献.doc

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Protective Effects of Human iPS-Derived Retinal Pigment Epithelium Cell Transplantation in the Retinal Dystrophic Rat 英文参考文献

ProtectiveEffectsofHumaniPS-DerivedRetinalPigment EpitheliumCellTransplantationintheRetinalDystrophic Rat Amanda-JayneCarr1.*,AnthonyA.Vugler1.,SherryT.Hikita2.,JeanM.Lawrence1.,CarlosGias1,LiLi Chen1,DavidE.Buchholz2,AhmadAhmado1,Ma’ayanSemo1,MatthewJ.K.Smart1,ShazeenHasan1, LyndondaCruz4,LincolnV.Johnson2,3,DennisO.Clegg2,3,PeteJ.Coffey1 1DepartmentofOcularBiologyandTherapeutics,InstituteofOphthalmology,UniversityCollegeLondon,London,UnitedKingdom,2CenterforStemCellBiologyand Engineering,DepartmentofMolecular,CellularandDevelopmental Biology,UniversityofCaliforniaSantaBarbara,SantaBarbara,California,UnitedStatesofAmerica, 3CenterfortheStudyofMacularDegeneration,UniversityofCaliforniaSantaBarbara,SantaBarbara,California,UnitedStatesofAmerica,4DepartmentofVitreoretinal Surgery,MoorfieldsEyeHospital,London,UnitedKingdom Abstract Transformationofsomaticcellswithasetofembryonictranscriptionfactorsproducescellswiththepluripotentproperties ofembryonicstemcells(ESCs).Theseinducedpluripotentstem(iPS)cellshavethepotentialtodifferentiateintoanycell type,makingthemapotentialsourcefromwhichtoproducecellsasatherapeuticplatformforthetreatmentofawide range of diseases. In many forms of human retinal disease, including age-related macular degeneration (AMD), the underlyingpathogenesisresideswithinthesupportcellsoftheretina,theretinalpigmentepithelium(RPE).Asamonolayer ofcellscriticaltophotoreceptorfunctionandsurvival,theRPEisanideallyaccessibletargetforcellulartherapy.Herewe report the differentiation of human iPS cells into RPE. We found that differentiated iPS-RPE cells were morphologically similar to, and expressed numerous markers of developing and mature RPE cells. iPS-RPE are capable of phagocytosing photoreceptormaterial,invitroandinvivofollowingtransplantationintotheRoyalCollegeofSurgeons(RCS)dystrophic rat.OurresultsdemonstratethatiPScellscanbedifferentiatedintofunctionaliPS-RPEandthattransplantationofthesecells canfacilitatetheshort-termmaintenanceofphotoreceptorsth