Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity AssaysDefining phenotype, tropism, and retinal gene therapy using adeno-associated viral vectors (AAVs) in new-born brown norway rats with a spontaneous mutation in CRB1Production of iPS-Derived Human Retinal Organoids for Use in Transgene Expression AssaysHuman 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 organoidsAAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotypeAAV Serotype Testing on Cultured Human Donor Retinal Explants
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Thilo M Buck