| Description | 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. |
| molecular weight | 342.37 |
| Molecular formula | C16H14N4O3S |
| CAS | 1459687-89-8 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
| Solubility | DMSO: 65 mg/mL (189.85 mM) |
| References | 1. Du L, et al. Mol Ther. 2013 Sep;21(9):1653-60. |
| Citations | 1. Perez H, Abdallah M F, Chavira J I, et al. A novel, ataxic mouse model of Ataxia Telangiectasia caused by a clinically relevant nonsense mutation. Elife. 2021, 10: e64695. |