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AAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype

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Purine metabolism was also significantly enriched, indicating compensatory remodeling of nucleotide pools in response to impaired pyrimidine biosynthesis

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FABP5 KO BMDMs were nucleofected with 5 g pXJ40-3xFlag-FABP5 WT or C127S plasmid using the Amaxa Mouse Macrophage Nucleofector Kit (Lonza, VPA-1009) following the manufacturers instructions