4A3-SC8
Instructions for Use
Product information
4A3-SC8 is a novel Ionizable amino lipid for RNA delivery.The CRISPR-Cas9 gene editing system has been a hotspot in the field of gene therapy, especially the gene correction induced by homology-directed repair (HDR). However, its application has various obstacles, such as large molecular weight, poor stability, off-target risk, and the complexity of codeliver multiple genes. Farbiak et al. established a novel ionizable lipid library consisting of four distinct amine cores (3A3, 3A5, 4A1, 4A3) and nine peripheries with different alkyl chain lengths (SC5-SC14), and screened out a class of iLNPs with ability of encapsulating Cas9 mRNA, sgRNA and donor DNA simultaneously. The delivery efficiency (quantified by luciferase mRNA expression) and iLNPs toxicity were evaluated with three different cell lines (HEK293T, HeLa, and IGROV-1), indicating the formulation containing 4A3-SC8 was the best. 4A3-SC8 iLNPs successfully induced HDR in HEK293 cells by one-pot delivery of Cas9 mRNA, sgRNA, and the correct ssDNA template. Confocal microscopy imaging showed that a portion of blue fluorescence in cells was corrected to green fluorescence. Furthermore, the nucleic acid ratios of Cas9: sgRNA: donor DNA loading in iLNPs at a ratio of 2:1:3 could maximize the HDR efficiency with the editing efficiency up to 23%, which breaks through the current bottleneck of HDR efficiency of only 1–5%. This progress is undoubtedly an important advance in the gene therapy field to cure diseases caused by genetic mutations.
| CAS No. | Not available |
| Chemical Name | 4A3-SC8 |
| Formula | C75H139N3O16S4 |
| M.Wt | 1,467.2 |
| 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: 1467.2 g/mol. Volumes are final solution volumes.
| Lipid mass | 1 mM | 5 mM | 10 mM |
|---|---|---|---|
| 10 mg | 6.816 mL | 1.363 mL | 0.682 mL |
| 25 mg | 17.039 mL | 3.408 mL | 1.704 mL |
| 50 mg | 34.079 mL | 6.816 mL | 3.408 mL |
| 100 mg | 68.157 mL | 13.631 mL | 6.816 mL |
| 250 mg | 170.393 mL | 34.079 mL | 17.039 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
1 Theranostic dendrimer-based lipid nanoparticles containing PEGylated BODIPY dyes for tumor imaging and systemic mRNA delivery in vivo-Journal of Controlled Release by Hu Xionga, Shuai Liua, Tuo Weia, Qiang Chenga, Daniel J. Siegwarta||||| 2 Preparation of selective organ-targeting (SORT) lipid nanoparticles (LNPs) using multiple technical methods for tissue-specific mRNA delivery--Nature Protocols volume 18, pages265–291 (2023)