GJ103 sodium salt is an active analog of the read-through compound GJ072. Chemical-induced read through of premature stop codons might be exploited as a potential treatment strategy for genetic disorders caused by nonsense mutations.
CAS Number: 1459687-96-7
Molecular Weight: 364.35
GJ 103 sodium
Chemical Name: sodium 2-((4-(3-methoxyphenyl)-5-(pyridin-2-yl)-4H-1,2,4-triazol-3-yl)thio)acetate
Appearance: Solid Power
Purity: ≥98% (or refer to the Certificate of Analysis)
Solubility: Soluble in DMSO
Shipping Condition: Shipped under ambient temperature as non-hazardous chemical or refer to Certificate of Analysis
Storage Condition: Dry, dark and -20 oC for 1 year or refer to the Certificate of Analysis.
Shelf Life: ≥12 months if stored properly.
Stock Solution Storage: 0 - 4 oC for 1 month or refer to the Certificate of Analysis.
Drug Formulation: To be determined.
HS Tariff Code: 382200
How to use
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.
GJ103 sodium salt is water soluble, making it much easier to work with in in vivo experiments.
- Du L, Jung ME, Damoiseaux R, Completo G, Fike F, Ku JM, Nahas S, Piao C, Hu H, Gatti RA. A new series of small molecular weight compounds induce read through of all three types of nonsense mutations in the ATM gene. Mol Ther. 2013 Sep;21(9):1653-60. doi: 10.1038/mt.2013.150. Epub 2013 Jun 18. PubMed PMID: 23774824; PubMed Central PMCID: PMC3776636.
- Lavin MF. Generating SM(a)RTer compounds for translation termination suppression in A-T and other genetic disorders. Mol Ther. 2013 Sep;21(9):1650-2. doi: 10.1038/mt.2013.177. PubMed PMID: 24008619; PubMed Central PMCID: PMC3776641.
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