The nucleoside analog clitocine is a potent and efficacious readthrough agent.

ABSTRACT: Nonsense mutations resulting in a premature stop codon in an open reading frame occur in critical tumor suppressor genes in a large number of the most common forms of cancers and are known to cause or contribute to the progression of disease. Low molecular weight compounds that induce readthrough of nonsense mutations offer a new means of treating patients with genetic disorders or cancers resulting from nonsense mutations. We have identified the nucleoside analog clitocine as a potent and efficacious suppressor of nonsense mutations. We determined that incorporation of clitocine into RNA during transcription is a prerequisite for its readthrough activity; the presence of clitocine in the third position of a premature stop codon directly induces readthrough. We demonstrate that clitocine c

SUBMITTER: Friesen WJ 

PROVIDER: S-EPMC5340919 | biostudies-literature | 2017 Apr

REPOSITORIES: biostudies-literature