Erratum to: A flow cytometry-based reporter assay identifies macrolide antibiotics as nonsense mutation read-through agents
2016
A large number of human diseases are caused by nonsense mutations. These mutations result in premature protein termination and the expression of truncated, usually nonfunctional products. A promising therapeutic strategy for patients suffering from premature termination codon (PTC)-mediated disorders is to suppress the nonsense mutation and restore the expression of the affected protein. Such a suppression approach using specific antibiotics and other read-through promoting agents has been shown to suppress PTCs and restore the production of several important proteins. Here, we report the establishment of a novel, rapid, and very efficient method for screening stop-codon read-through agents. We also show that,inbothmammaliancellsandinatransgenicmousemodel, distinct members of the macrolide antibiotic family can induce read-through of disease-causing stop codons leading to reexpression of several key proteins and to reduced disease
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