TargetMol

RTC13

Product Code:
 
TAR-T34425
Product Group:
 
Inhibitors and Activators
Supplier:
 
TargetMol
Regulatory Status:
 
RUO
Shipping:
 
cool pack
Storage:
 
-20℃
1 / 1
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Further Information

Bioactivity:
RTC13, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy.
CAS:
1359825-94-7
Formula:
C14H9N3O4S
Molecular Weight:
315.3
Purity:
0.98
SMILES:
[O-][N+](=O)c1ccccc1-c1ccc(C=C2/SC(=N)NC2=O)o1

References

G?mez-Grau M, Garrido E, Cozar M, Rodriguez-Sureda V, Dom?nguez C, Arenas C, Gatti RA, Cormand B, Grinberg D, Vilageliu L. Evaluation of Aminoglycoside and Non-Aminoglycoside Compounds for Stop-Codon Readthrough Therapy in Four Lysosomal Storage Diseases. PLoS One. 2015 Aug 19;10(8):e0135873. doi: 10.1371/journal.pone.0135873. eCollection 2015. PubMed PMID: 26287674; PubMed Central PMCID: PMC4545610. Nakamura K, Du L, Tunuguntla R, Fike F, Cavalieri S, Morio T, Mizutani S, Brusco A, Gatti RA. Functional characterization and targeted correction of ATM mutations identified in Japanese patients with ataxia-telangiectasia. Hum Mutat. 2012 Jan;33(1):198-208. doi: 10.1002/humu.21632. Epub 2011 Nov 9. PubMed PMID: 22006793; PubMed Central PMCID: PMC3261637. Kayali R, Ku JM, Khitrov G, Jung ME, Prikhodko O, Bertoni C. Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy. Hum Mol Genet. 2012 Sep 15;21(18):4007-20. doi: 10.1093/hmg/dds223. Epub 2012 Jun 12. PubMed PMID: 22692682; PubMed Central PMCID: PMC3607466. Du L, Jung ME, Damoiseaux R, Completo G, Fike F, Ku JM, Nahas S, Piao C, Hu H, Gatti RA. A new series of small molecular weight compounds induce read through of all three types of nonsense mutations in the ATM gene. Mol Ther. 2013 Sep;21(9):1653-60. doi: 10.1038/mt.2013.150. Epub 2013 Jun 18. PubMed PMID: 23774824; PubMed Central PMCID: PMC3776636.