Ataluren
Product Specifications
UNSPSC Description
Ataluren (PTC124) is an orally available CFTR-G542X nonsense allele inhibitor.
Target Antigen
Autophagy; CFTR
Type
Reference compound
Related Pathways
Autophagy;Membrane Transporter/Ion Channel
Applications
Metabolism-protein/nucleotide metabolism
Field of Research
Metabolic Disease; Cancer
Assay Protocol
https://www.medchemexpress.com/PTC124.html
Solubility
DMSO : ≥ 52 mg/mL
Smiles
O=C(C1=CC=CC(C2=NOC(C3=CC=CC=C3F)=N2)=C1)O
Molecular Weight
284.24
References & Citations
[1]Pettit RS, et al. CFTR Modulators for the Treatment of Cystic Fibrosis. P T. 2014 Jul;39(7):500-11.|[2]Welch EM, et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature, 2007, 447(7140), 87-91.|[3]Miller JN, et al. The novel Cln1(R151X) mouse model of infantile neuronal ceroid lipofuscinosis (INCL) for testing nonsense suppression therapy. Hum Mol Genet. 2015 Jan 1;24(1):185-96.
Shipping Conditions
Room Temperature
Product Datasheet
http://file.medchemexpress.com/batch_PDF/HY-14832/Ataluren-DataSheet-MedChemExpress.pdf
Product MSDS
http://file.medchemexpress.com/batch_PDF/HY-14832/Ataluren-SDS-MedChemExpress.pdf
Clinical Information
Launched
CAS Number
775304-57-9
Available Sizes
Curated Selection
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