5A2-SC8
Instructions for Use
Product information
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 |
| 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. |
Formulation & study context
Literature information refers to the cited study and its particular formulation. Administration routes are research context, not clinical instructions or a guarantee of performance.
A complete product-specific formulation has not been verified. No standard ratio is substituted.
| N/P ratio | Not reported in the verified record |
| Cargo | Not available in the verified record |
| Administration route | Not available in the verified record |
| Animal model | Not available in the verified record |
| Formulation process | Not available in the verified record |
N/P is the molar ratio of specified lipid nitrogen groups to nucleic-acid phosphate groups. A lipid:cargo mass ratio is a different quantity and must not be used as N/P.
Stock-solution preparation table
Calculated from the product-page molecular weight: 1841.72 g/mol. Volumes are final solution volumes.
| Lipid mass | 1 mM | 5 mM | 10 mM |
|---|---|---|---|
| 10 mg | 5.430 mL | 1.086 mL | 0.543 mL |
| 25 mg | 13.574 mL | 2.715 mL | 1.357 mL |
| 50 mg | 27.149 mL | 5.430 mL | 2.715 mL |
| 100 mg | 54.297 mL | 10.859 mL | 5.430 mL |
| 250 mg | 135.743 mL | 27.149 mL | 13.574 mL |
Volume (mL) = mass (mg) × 1000 / [MW (g/mol) × concentration (mM)]. These are mathematical conversions, not measured solubility or validated solvent recommendations. Confirm solvent compatibility and the molecular weight of the exact material before use.
Open editable solution calculators →Handling & storage
Provided that storage conditions are as stated on the product vial and the vial is kept tightly sealed, the product can be stored for up to 1 year.
Wherever possible, prepare and use solutions on the same day. If stock solutions must be prepared in advance, store them as aliquots in tightly sealed vials at -20°C. Generally, these will be usable for up to one month.
Before use, and prior to opening the vial, allow the product to equilibrate to room temperature for at least 1 hour.
References
Yehui Sun et al. In vivo editing of lung stem cells for durable gene correction in mice. Science, 2024, doi:10.1126/science.adk9428.