The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential.

ABSTRACT: Nonsense mutations, resulting in a premature stop codon in the open reading frame of mRNAs are responsible for thousands of inherited diseases. Readthrough of premature stop codons by small molecule drugs has emerged as a promising therapeutic approach to treat disorders resulting from premature termination of translation. The aminoglycoside antibiotics are a class of molecule known to promote readthrough at premature termination codons. Gentamicin consists of a mixture of major and minor aminoglycoside components. Here, we investigated the readthrough activities of the individual components and show that each of the four major gentamicin complex components representing 92-99% of the complex each had similar potency and activity to that of the complex itself. In contrast, a minor component

SUBMITTER: Friesen WJ 

PROVIDER: S-EPMC6201930 | biostudies-literature | 2018

REPOSITORIES: biostudies-literature