Mechanism and evidence of nonsense suppression therapy for genetic eye disorders.

Abstract

Between 5 and 70% of genetic disease is caused by in-frame nonsense mutations, which introduce a premature termination codon (PTC) within the disease-causing gene. Consequently, during translation, non-functional or gain-of-function truncated proteins of pathological significance, are formed. Approximately 50% of all inherited retinal disorders have been… (More)
DOI: 10.1016/j.exer.2017.01.001

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