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.