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Targeted ablation of Crb1 and Crb2 in retinal progenitor cells mimics Leber Congenital Amaurosis.Defining phenotype, tropism, and retinal gene therapy using adeno-associated viral vectors (AAVs) in new-born brown norway rats with a spontaneous mutation in CRB1PSD95beta regulates plasma membrane Ca(2+) pump localization at the photoreceptor synapse.Gene therapy into photoreceptors and Müller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse modelsMicroarray and morphological analysis of early postnatal CRB2 mutant retinas on a pure C57BL/6J genetic background.Specific tools for targeting and expression in Müller glial cellsHuman iPSC-Derived Retinas Recapitulate the Fetal CRB1 CRB2 Complex Formation and Demonstrate that Photoreceptors and Müller Glia Are Targets of AAV5AAV-CRB2 protects against vision loss in an inducible CRB1 retinitis pigmentosa mouse modelCRB1 is required for recycling by RAB11A+ vesicles in human retinal organoidsGFAP-driven GFP expression in activated mouse Muller glial cells aligning retinal blood vessels following intravitreal injection of AAV2/6 vectors.Targeted ablation of Crb2 in photoreceptor cells induces retinitis pigmentosaAAV Serotype Testing on Cultured Human Donor Retinal ExplantsCRB2 acts as a modifying factor of CRB1-related retinal dystrophies in mice
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Public - No restriction
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Publiek - Geen beperking
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