Lipid 8
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Ionizable lipid for LNP and nucleic-acid delivery research.
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.
| 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 | Pure form: −20 °C, 1 year; In solvent: −80 °C, 6 months; −20 °C, 1 month. Sealed and protected from light. |
| 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. |
The product molecular weight is prefilled when available and remains editable. Confirm solvent compatibility and solubility before preparation.
Solvent volumes update from the molecular weight above.
| Mass | 1 mM | 5 mM | 10 mM |
|---|---|---|---|
| 10 mg | 5.4297 mL | 1.0859 mL | 542.97 μL |
| 25 mg | 13.5743 mL | 2.7149 mL | 1.3574 mL |
| 50 mg | 27.1485 mL | 5.4297 mL | 2.7149 mL |
| 100 mg | 54.2971 mL | 10.8594 mL | 5.4297 mL |
| 250 mg | 135.7427 mL | 27.1485 mL | 13.5743 mL |
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