5A2-SC8
Version 1.3e1167b1 · 2026-09-13
| CAS No. | Not available |
| Chemical Name | 5A2-SC8 |
| Synonyms | 5A2SC8， 5A2 SC8 |
| SMILES | C(OCCOC(=O)C(C)CSCCCCCCCC)(=O)CCN(CCC(OCCOC(=O)C(C)CSCCCCCCCC)=O)CCNCCN(CCC(OCCOC(=O)C(C)CSCCCCCCCC)=O)CCNCCN(CCC(OCCOC(=O)C(C)CSCCCCCCCC)=O)CCC |
| Formula | C93H173N5O20S5 |
| M.Wt | 1,841.72 |
| Purity | ELSD-HPLC>95% |
| Storage | Original product: up to 1 year under the storage conditions stated on the vial, kept tightly sealed. Stock solutions: aliquots at -20°C, generally up to one month. |
| Shipping condition | Ships from Shanghai. Pure lipid is stable during ice-pack transport. |
| Publication | Yehui Sun et al. In vivo editing of lung stem cells for durable gene correction in mice. Science, 2024, doi:10.1126/science.adk9428. |
5A2-SC8 is a dendrimer for miRNA delivery to late-stage liver tumors with low hepatotoxicity. 5A2-SC8 shows potent EC50 < 0.02 mg/kg (siRNA against FVII (siFVII)) in dose-response experiments, and well tolerated in separate toxicity studies in chronically ill mice bearing MYC-driven tumors. 5A2-SC8 is a degradable lipid-like compound (ester-based dendrimer) for small RNAs delivery.5A2-SC8, was obtained by screening a large library of more than 1500 ester-based dendrimers containing ionizable amino groups, which have three tertiary amine heads and five lipid tails. Based on this library, the in vitro transfection efficiency of different formulations of 5A2-SC8 iLNPs was evaluated, discovering the optimal formulation (5A2-SC8, DOPE, cholesterol, PEG at a molar ratio of 15:15:30:3) of 5A2-SC8 iLNPs for delivering fumarylacetoacetate hydrolase (FAH) mRNA to liver.After the intravenous injection via tail, the model mice of hepatorenal tyrosinemia type I had strong FAH protein expression, which prevented body weight loss and increased the survival rate of hepatorenal tyrosinemia mice . In addition to introducing utility of 5A2-SC8 iLNPs for the therapeutic intervention, the 5A2-SC8 iLNPs containing DOTAP have been used to establish complex mouse models via intravenous injection, including in situ liverspecific cancer model and in situ lung-specific cancer model. Based on this iLNPs delivery system, 5A2-SC8 induced model construction method overcomes the time-consuming and costly disadvantages of traditional animal models establishing methods, including transgenesis and gene engineering in embryonic stem cells.
Specifications shown are product-page values, not batch test results.
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