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Apexbio Technology LLC PTC124 (Ataluren) 775304-57-9 10mM (in 1mL DMSO)
Supplier: Apexbio Technology LLC A85535.1
PTC124 (Ataluren CAS 775304-57-9) is a small molecule modulator that selectively targets premature termination codons (nonsense mutations) which are point mutations causing early termination during translation By promoting ribosomal read-through of these mutations PTC124 enhances the synthesis of full-length functional proteins In vitro studies demonstrated its activity at IC50 of approximately 0 1 M Research in MDX mouse skeletal muscle cells and human induced pluripotent stem cell-derived retinal pigment epithelium cells confirmed its capacity to restore protein production Animal models of Duchenne muscular dystrophy and cystic fibrosis exhibited improved muscle and channel protein function upon oral or subcutaneous administration suggesting its potential for addressing genetic diseases associated with nonsense mutations
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