Field of application
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.: |
1857341-90-2 |
| Chemical Name: |
NND-22111102 |
| 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)CC |
| Formula: |
C93H173N5O20S5 |
| M.Wt: |
1841.72 |
| Purity: |
>95% |
| Sotrage: |
2 years -20°C Powder, 2 weeks 4°C in DMSO, 6 months -80°C in DMSO |
