Defining phenotype, tropism, and retinal gene therapy using adeno-associated viral vectors (AAVs) in new-born brown norway rats with a spontaneous mutation in CRB1CRB2 Loss in Rod Photoreceptors Is Associated with Progressive Loss of Retinal Contrast SensitivitymRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapyCrumbs2 Is an Essential Slit Diaphragm Protein of the Renal Filtration BarrierCharacterization and AAV-mediated CRB gene augmentation in human-derived CRB1KO and CRB1KOCRB2+/- retinal organoidsCRB1 is required for recycling by RAB11A+ vesicles in human retinal organoidsAAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype
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Nanda Boon