PTC124 (Ataluren) Chemical Structure
[Ala92]-p16 (84-103) is a CDK inhibitor with IC50 of 1.5μM for cyclin-dependent kinase-4 (cdk4)/cyclin D1
Bax inhibitor peptide,negative control is a negative control peptide for the Bax inhibitor peptides V5 and P5.
JNK and p38 MAPK inhibitor
SRC kinase inhibitor
Targeting VHL-deficient RCC cells, in renal cell carcinoma (RCC) cells that have lost the von Hippel-Lindau (VHL) tumor suppressor activity (IC50 = 625 μM).
BTZ043 racemate is a decaprenylphosphoryl-b-D-ribose 2’-epimerase inhibitor with MIC of 2.3 and 9.2 nM for M. tuberculosis H37Rv and Mycobacterium smegmatis, respectively.
GSK690693 is a pan-Akt kinase inhibitor,IC50 of Akt 1, 2, and 3 at 2, 13, and 9 nM respectively.
cathepsin K inhibitor with an IC50 of 0.2nM.
Malotilate is a liver protein metabolism improved compound.
PA-824 is an experimental anti-tuberculosis drug with MIC of less than 1 μg/mL for tuberculosis.
PTC124 targets genetic disorders caused by nonsense mutations. [1] PTC124 is an orally bioavailable compound that promotes of the human CFTR-G542X nonsense allele in a CF mouse model. [2] Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial. [3]
PTC124 induces dose-dependent suppression of premature translational termination without concomitant effects on normal termination or mRNA decay. [1]
In safety pharmacology studies in rats and dogs, oral administration of PTC124 induces no adverse neurological, pulmonary, or cardiovascular effects at doses through 1500 mg/kg. In toxicology studies in rats and dogs at oral doses through 1500 mg/kg for 28 days, PTC124 has shown good tolerability. [1]
The minimal concentration of PTC124 showing discernable readthrough was 0.01–0.1 mM (2.8–28 ng ml-1), whereas the concentration promoting maximal activity was approximately 3 mM(852 ng ml-1). [1]
| Molecular Weight (WM): | 284.24 |
|---|---|
| Formula: | C15H9FN2O3 |
| CAS No.: | 775304-57-9 |
| Synonyms: |
N/A
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| Dissolve in (25°C): | DMSO ≥57mg/mL |
| Water <1mg/mL | |
| Ethanol <1mg/mL | |
| Storage: | 2 years-20°CPowder |
| 1 week-4°Cin DMSO | |
| 1 month-80°in DMSO |
A collection of 864 bioactive compounds
A collection of 481 inhibitors
A collection of 194 kinase inhibitors
A collection of 85 tyrosine kinase inhibitors.
A collection of 426 FDA approved drugs
A collection of 139 natural products
A collection of 40 chemotherapeutic agents
A unique collection of 17 small molecule modulators
A unique collection of 47 small molecule inhibitors
A unique collection of 63 GPCR small molecules

Quantitative RT-PCR analysis of CPT1A in normal and patient fibroblasts treated with various concentration of PTC124 (0, 1, 3 and 5 μm) for 72 hours. The relative contents of mRNA were calculated by using the ΔΔCt method. Expression was normalized to human GAPDH and no significant fold difference was observed in the patient mRNA levels after treatment with PTC124. The normal control cells showed approximately 10-fold higher expression than patient cells. Data are presented as mean; Error bar indicates SEM |
Quantitative RT-PCR analysis of CPT1A in normal and patient fibroblasts treated with various concentration of PTC124 (0, 1, 3 and 5 μm) for 72 hours. The relative contents of mRNA were calculated by using the ΔΔCt method. Expression was normalized to human GAPDH and no significant fold difference was observed in the patient mRNA levels after treatment with PTC124. The normal control cells showed approximately 10-fold higher expression than patient cells. Data are presented as mean; Error bar indicates SEM
Data independently produced by Dr Lu Tan of Children’s Hospital of Philadelphia PTC124 (Ataluren) purchased from Selleck

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