化合物信息

Description

Inflammation

Assays for read-through of LUC premature termination codons

Stock solutions of PTC124 (6 mM) are prepared in 100% DMSO HEK293 cells grown in a medium containing fetal bovine serum (FBS) and are stably transfected with LUC genes containing premature terminators at codon 190 (and 11 contextual variants thereof), treated with increasing concentrations of PTC124 for 16 hours, and assayed for luciferase activity. Luminescence of each culture relative to the control is quantified by standard procedures using a Viewlux CCD imager. Luminescence data are normalized to that produced with DMSO, and the fold suppression over background is calculated as (PTC124_{light units}/DMSO_{light units}).

Animal Models

Cftr-/- hCFTR-G542X transgenic mice

Formulation

Dissolved in DMSO, and diluted in saline

Doses

~60 mg/kg/day

Administration

Subcutaneous injection or oral administration