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.
| Related Products |
|---|
| M3554 (anti-GD2 ADC)
M3554 is a novel anti-GD2 ADC designed based on humanized ch14.18 anti-GD2 antibody. M3554 has anti-tumor activity. |
| OGT2115
OGT2115 is a potent, cell-permeable and orally active heparanase inhibitor with an IC50 of 0.4 μM. OGT2115 has anti-angiogenic properties (IC50 = 1 μM). |
| DiosMetin 7-O-β-D-Glucuronide
DiosMetin 7-O-β-D-Glucuronide is an antioxidant constituent in the fruits of Luffa cylindrical. |
| Bendazac L-Lysine
Bendazac L-Lysine is an aldose reductase (AR) inhibitor, which can be used to inhibit the activity of AR in the eye to prevent cataracts. |
| Ro5-3335
Ro5-3335 acts as an inhibitor of core binding factor (CBF) leukemia. Ro5-3335 is a RUNX1-CBFβ interaction inhibitor that represses RUNX1/CBFB-dependent transactivation. |
Products are for research use only. Not for human use. We do not sell to patients.
© Copyright 2010-2023 AbMole BioScience. All Rights Reserved.
