GJ103

GJ103 sodium salt is an active analog of the read-through compound GJ072. Some GJ072 analogs (e.g., GJ103, GJ106, GJ109, and GJ111) consistently demonstrates their activities in all three PTCs by both FCATMpSer1981 and IRIF assays. GJ103 have similar read-through activity as RTC13 or RTC14, but are more tolerable than RTC13 and RTC14 to A-T cells.