Ataluren
Ataluren (PTC124) is an orally available CFTR-G542X nonsense allele inhibitor.
Product Specifications
CAS Number
[775304-57-9]
Product Name Alternative
PTC124
UNSPSC
12352005
Hazard Statement
H302, H315, H319, H335
Target
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
Purity
99.62
Solubility
DMSO : ≥ 52 mg/mL
Smiles
O=C(C1=CC=CC(C2=NOC(C3=CC=CC=C3F)=N2)=C1)O
Molecular Formula
C15H9FN2O3
Molecular Weight
284.24
Precautions
H302, H315, H319, H335
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
Storage Conditions
-20°C, 3 years; 4°C, 2 years (Powder)
Scientific Category
Reference compound1
Clinical Information
Launched
Available Sizes
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