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dc.rights.licenseopenen_US
dc.contributor.authorKOLE, C.
dc.contributor.authorKLIPFEL, L.
dc.contributor.authorYANG, Y.
dc.contributor.authorFERRACANE, V.
dc.contributor.authorBLOND, F.
dc.contributor.authorREICHMAN, S.
dc.contributor.authorMILLET-PUEL, G.
dc.contributor.authorCLERIN, E.
dc.contributor.authorAIT-ALI, N.
dc.contributor.authorPAGAN, D.
dc.contributor.authorCAMARA, H.
hal.structure.identifierBordeaux population health [BPH]
dc.contributor.authorDELYFER, Marie-Noelle
dc.contributor.authorNANDROT, E. F.
dc.contributor.authorSAHEL, J. A.
dc.contributor.authorGOUREAU, O.
dc.contributor.authorLEVEILLARD, T.
dc.date.accessioned2020-11-23T12:27:32Z
dc.date.available2020-11-23T12:27:32Z
dc.date.issued2018-01-03
dc.identifier.issn1525-0016en_US
dc.identifier.urihttps://oskar-bordeaux.fr/handle/20.500.12278/15571
dc.description.abstractEnInherited retinal degenerations are blinding diseases characterized by the loss of photoreceptors. Their extreme genetic heterogeneity complicates treatment by gene therapy. This has motivated broader strategies for transplantation of healthy retinal pigmented epithelium to protect photoreceptors independently of the gene causing the disease. The limited clinical benefit for visual function reported up to now is mainly due to dedifferentiation of the transplanted cells that undergo an epithelial-mesenchymal transition. We have studied this mechanism in vitro and revealed the role of the homeogene OTX2 in preventing dedifferentiation through the regulation of target genes. We have overexpressed OTX2 in retinal pigmented epithelial cells before their transplantation in the eye of a model of retinitis pigmentosa carrying a mutation in Mertk, a gene specifically expressed by retinal pigmented epithelial cells. OTX2 increases significantly the protection of photoreceptors as seen by histological and functional analyses. We observed that the beneficial effect of OTX2 is non-cell autonomous, and it is at least partly mediated by unidentified trophic factors. Transplantation of OTX2-genetically modified cells may be medically effective for other retinal diseases involving the retinal pigmented epithelium as age-related macular degeneration.
dc.language.isoENen_US
dc.rightsAttribution-NonCommercial-NoDerivs 3.0 United States*
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/3.0/us/*
dc.subject.enLEHA
dc.title.enOtx2-Genetically Modified Retinal Pigment Epithelial Cells Rescue Photoreceptors after Transplantation
dc.title.alternativeMol Theren_US
dc.typeArticle de revueen_US
dc.identifier.doi10.1016/j.ymthe.2017.09.007en_US
dc.subject.halSciences du Vivant [q-bio]/Santé publique et épidémiologieen_US
dc.identifier.pubmed28988713en_US
bordeaux.journalMolecular therapyen_US
bordeaux.page219-237en_US
bordeaux.volume26en_US
bordeaux.hal.laboratoriesBordeaux Population Health Research Center (BPH) - U1219en_US
bordeaux.issue1en_US
bordeaux.institutionUniversité de Bordeauxen_US
bordeaux.teamLEHA_BPH
bordeaux.teamLEHA_BPH
bordeaux.peerReviewedouien_US
bordeaux.inpressnonen_US
hal.exportfalse
bordeaux.COinSctx_ver=Z39.88-2004&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.jtitle=Molecular%20therapy&rft.date=2018-01-03&rft.volume=26&rft.issue=1&rft.spage=219-237&rft.epage=219-237&rft.eissn=1525-0016&rft.issn=1525-0016&rft.au=KOLE,%20C.&KLIPFEL,%20L.&YANG,%20Y.&FERRACANE,%20V.&BLOND,%20F.&rft.genre=article


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