A nonaminoglycoside that has been reported to selectively induce ribosomes to read through premature nonsense stop signals on mRNA

PTC-124 – 5 mg

Brand:
Cayman
CAS:
775304-57-9
Storage:
-20
UN-No:
Non-Hazardous - /

Nonsense mutations create a premature termination of mRNA translation and have been implicated in various genetic disorders, including muscular dystrophy and cystic fibrosis.{27189} PTC-124 is a nonaminoglycoside that has been reported to selectively induce ribosomes to read through premature nonsense stop signals on mRNA, thus allowing the production of full length, functional proteins.{27189} In a mouse model of cystic fibrosis caused by nonsense mutations, PTC-124 treatment (60 mg/kg s.c. injection or 0.3-0.9 mg/ml orally) has been shown to restore cystic fibrosis transmembrane conductance regulator (CFTR) protein expression and function.{27187} The target activity of PTC-124 was initially evaluated by firefly luciferase reporter cell-based nonsense codon assay (IC50 = 7 nM); however, subsequent assessments using a Renilla reniformis luciferase reporter have failed to produce nonsense codon suppression activity.{27188} Thus, while PTC-124 is in clinical testing in patients with nonsense mutations within the CFTR or dystrophin genes, controversy surrounds its exact mechanism of action.{27189,27188,27191,27190}  

 

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Description

A nonaminoglycoside that has been reported to selectively induce ribosomes to read through premature nonsense stop signals on mRNA, thus allowing the production of full length, functional proteins (IC50 = 7 nM); 60 mg/kg s.c. injection or 0.3-0.9 mg/ml orally has been shown to restore CFTR protein expression and function in a mouse model of cystic fibrosis caused by nonsense mutations

Formal name: 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]-benzoic acid

Synonyms:  Ataluren

Molecular weight: 284.3

CAS: 775304-57-9

Purity: ≥98%

Formulation: A crystalline solid

Product Type|Biochemicals||Research Area|Neuroscience

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