Dual CRALBP isoforms unveiled: iPSC-derived retinal modeling and AAV2/5-RLBP1 gene transfer raise considerations for effective therapy.

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作者： Krishna,Damodar ^[1] ; Gregor,Dubois ^[1] ; Laurent,Guillou ^[1] ; Daria,Mamaeva ^[1] ; Marie,Pequignot ^[1] ; Nejla,Erkilic ^[2] ; Carla,Sanjurjo-Soriano ^[1] ; Hassan,Boukhaddaoui ^[1] ; Florence,Bernex ^[3] ; Béatrice,Bocquet ^[2] ; Jérôme,Vialaret ^[4] ; Yvan,Arsenijevic ^[5] ; T Michael,Redmond ^[6] ; Christopher,Hirtz ^[4] ; Isabelle,Meunier ^[2] ; Philippe,Brabet ^[1] ; Vasiliki,Kalatzis ^[7]

作者单位： Institute for Neurosciences of Montpellier (INM), University of Montpellier, Inserm, 34091 Montpellier, France. ^[1] Institute for Neurosciences of Montpellier (INM), University of Montpellier, Inserm, 34091 Montpellier, France; National Reference Centre for Inherited Sensory Diseases, University of Montpellier, Montpellier University Hospital, 34090 Montpellier, France. ^[2] RHEM, Réseau d'Histologie Expérimentale de Montpellier, University of Montpellier, Biocampus, CNRS, Inserm, 34298 Montpellier, France. ^[3] Institute for Neurosciences of Montpellier (INM), University of Montpellier, Inserm, 34091 Montpellier, France; IRMB-PPC, INM, Montpellier University Hospital, Inserm, CNRS, University of Montpellier, 34295 Montpellier, France. ^[4] Unit of Retinal Degeneration and Regeneration, Department of Ophthalmology, University Lausanne, Jules-Gonin Eye Hospital, Fondation Asile des Aveugles, 1004 Lausanne, Switzerland. ^[5] Laboratory of Retinal Cell and Molecular Biology, National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA. ^[6] Institute for Neurosciences of Montpellier (INM), University of Montpellier, Inserm, 34091 Montpellier, France; National Reference Centre for Inherited Sensory Diseases, University of Montpellier, Montpellier University Hospital, 34090 Montpellier, France. Electronic address: vasiliki.kalatzis@inserm.fr. ^[7]

关键词 AAV CRALBP RLBP1 RPE iPSC-derived RPE iPSC-derived retinal organoids inherited retinal diseases internal start codon protein isoforms visual cycle