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Failure to do so will result in return of the anemia and in development of incapacitating and irreversible damage to the nerves of the spinal cord

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Cell viability was measured by CCK-8 assay

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QR-110, an ASO drug in Phase III trials, was designed to address Leber congenital amaurosis type 10 caused by the p.Cys998X mutation in the centrosomal protein 290 gene