RTC13

RTC13 is a small molecule read-through reagent that promotes ribosomal read-through of premature termination codons (PTCs), enabling partial restoration of truncated protein expression in genetic diseases caused by nonsense mutations. In preclinical studies, RTC13 restored dystrophin protein expression in multiple muscle groups, including the diaphragm and heart, and improved muscle function and serum creatine kinase levels in the mdx mouse model of Duchenne muscular dystrophy (DMD). It is more effective than other read-through compounds such as gentamicin and PTC124 in this context. The mechanism involves interference with ribosomal translation, specifically enabling translational bypass of premature stop codons while sparing normal protein termination. RTC13 has been developed by researchers at the University of California, Los Angeles and is being investigated for other disorders caused by nonsense mutations[1][3][5][7].

Other names

WAY301503WAY-301503WAY 301503CAS# 313530-30-2