Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies
Sign Up to like & getrecommendations! Published in 2021 at "Expert Opinion on Investigational Drugs"
DOI: 10.1080/13543784.2021.1868434
Abstract: ABSTRACT Introduction Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused by mutations in the dystrophin (DMD) gene. Most patients die from respiratory failure or cardiomyopathy. There are significant unmet needs for treatments for… read more here.
Keywords: exon skipping; codon readthrough; gene replacement; stop codon ... See more keywords
UGA stop codon readthrough to translate intergenic region of Plautia stali intestine virus does not require RNA structures forming internal ribosomal entry site.
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DOI: 10.1261/rna.065466.117
Abstract: The translation of capsid proteins of Plautia stali intestine virus (PSIV), encoded in its second open reading frame (ORF2), is directed by an internal ribosomal entry site (IRES) located in the intergenic region (IGR). Owing… read more here.
Keywords: igr; codon readthrough; rna; codon ... See more keywords
Stop codon readthrough contexts influence reporter expression differentially depending on the presence of an IRES
Sign Up to like & getrecommendations! Published in 2020 at "Wellcome Open Research"
DOI: 10.12688/wellcomeopenres.16231.3
Abstract: Abstract Background: Previously we reported the discovery of stop codon readthrough in AMD1 mRNA followed by ribosome stalling at the end of a conserved Open Reading Frame (ORF) that we termed AMD1. To explain the… read more here.
Keywords: stop codon; codon readthrough; expression; reporter ... See more keywords