In vitro: Chemical-induced read through of premature stop codons might be exploited as a potential treatment strategy for genetic disorders caused by nonsense mutations. GJ072 is a novel read-through compound (RTC). GJ072 shows activity comparable to stop codons (TGA, TAG, and TAA) PTC124 and RTC13. GJ072 induces ATM kinase on both TGA and TAG stop codons and restored ATMpSer1981 autophosphorylation and SMC1pSer966 transphosphorylation as measured by FACS. GJ072 is active in A-T cells with a homozygous TAA mutation. GJ072 is able to induce detectable full-length ATM protein in treated A-T cells. Early structure-activity relationship studies generates eight active analogs of 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. GJ071 and GJ072 and some of their analogs (such as GJ103) have similar read-through activity as RTC13 or RTC14, but are more tolerable than RTC13 and RTC14 to A-T cells. GJ103 does not show obvious cytotoxicity in A-T cells at concentration as high as 300 μM.
In vivo: GJ103 sodium salt is water soluble, making it much easier to work with in in vivo experiments.
| Molecular Weight | 364.35 |
| Formula | C16H13N4NaO3S |
| CAS Number | 1459687-96-7 |
| Solubility (25°C) | 34 mg/mL in DMSO |
| Storage |
Powder -20°C 3 years ; 4°C 2 years In solvent -80°C 6 months ; -20°C 1 month |
| Species | Mouse | Rat | Rabbit | Guinea pig | Hamster | Dog |
| Weight (kg) | 0.02 | 0.15 | 1.8 | 0.4 | 0.08 | 10 |
| Body Surface Area (m2) | 0.007 | 0.025 | 0.15 | 0.05 | 0.02 | 0.5 |
| Km factor | 3 | 6 | 12 | 8 | 5 | 20 |
| Animal A (mg/kg) = Animal B (mg/kg) multiplied by | Animal B Km |
| Animal A Km |
For example, to modify the dose of Compound A used for a mouse (20 mg/kg) to a dose based on the BSA for a rat, multiply 20 mg/kg by the Km factor for a mouse and then divide by the Km factor for a rat. This calculation results in a rat equivalent dose for Compound A of 10 mg/kg.
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