nonsense

GJ071 oxalate is a Nonsense suppressor. Nonsense suppressors (NonSups) induce "readthrough", i.e., the selection of near cognate tRNAs at premature termination codons and insertion of the corresponding amino acid into nascent polypeptide.

ELX-02 disulfate is a synthetic eukaryotic ribosome-specific glycoside (ERSG) that induces translation read-through of nonsense mutations, producing normally localized, full-length functional proteins. It can be used to study genetic diseases caused by nonsense mutations.

Ataluren (PTC124) is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk.

eIF4A3-IN-1 is a selective and potent inhibitor of eukaryotic initiation factor 4A3 (eIF4A3) with an IC50 value of 0.26 μM. eIF4A3-IN-1 inhibits cytosolic nonsense-mediated RNA decay at high concentrations.

NMDI14 is a highly specific, small-molecule, non-competitive inhibitor of the nonsense-mediated RNA decay (NMD) pathway with an IC₅₀ value of 1.8 μM. NMDI14 can be used for studying NMD pathway function, PTC-related genetic disorders, and tumor research.

ELX-02 sulfate, also known as NB-124 sulfate, is a novel compound currently under development as a therapeutic agent for genetic diseases triggered by nonsense mutations. This investigational compound, an advanced synthetic eukaryotic ribosome selective glycoside (ERSG), shows promising potential for targeted treatment.

NV914 is an inhibitor of FTSJ1 (tryptophan tRNA-specific 2'-O-methyltransferase) that blocks the methyltransferase activity of FTSJ1. It induces the readthrough of premature stop codons, thereby allowing the synthesis of full-length proteins from genes carrying nonsense mutations. As a translation readthrough inducing compound (TRID), NV914 demonstrates readthrough activity for nonsense mutations in vitro without affecting natural stop codons, thus restoring CFTR protein expression. In mice, NV914 shows good acute oral tolerance, presenting low health risks and demonstrating favorable safety. NV914 is applicable in research related to cystic fibrosis and Shwachman-Diamond syndrome.

Clitocine, an adenosine analogue derived from mushrooms, is a potent transcriptional readout agent, an inhibitor of nonsense mutations, and has anticancer activity, inducing the production of p53 protein in cells carrying the p53 nonsense mutation allele.Clitocine enhances TRAIL-mediated apoptosis in human colon cancer cells by promoting Mcl-1 degradation.NSC 81464 is an alkaloid of the yohimbine type extracted from the roots and leaves of Rauwolfia vomitoria.

ELX-02, also known as NB-124, is a eukaryotic ribosomal selective glycoside (ERSG) designed to increase the read-through activity in patients with nonsense mutations and enable the production of sufficient amounts of full-length functional protein to rest

GJ-072 is a Nonsense suppressor. Nonsense suppressors (NonSups) induce "readthrough", i.e., the selection of near cognate tRNAs at premature termination codons and insertion of the corresponding amino acid into nascent polypeptide.

DMNB-caged-Serine is a photocaged amino acid, with DMNB as a blue light-sensitive caging group. It can be incorporated into proteins in Saccharomyces cerevisiae in response to the amber nonsense codon (TAG), facilitating control over the chemical composition of mammalian proteins.