5A2-SC8
Cat. No.: DC49907
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Chemical Structure
1857341-90-2
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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 |
Synonyms: |
4,7,10,13,16-Pentaazanonadecanedioic acid, 4,10,16-tris[3-[2-[2-methyl-3-(octylthio)-1-oxopropoxy]ethoxy]-3-oxopropyl]-, 1,19-bis[2-[2-methyl-3-(octylthio)-1-oxopropoxy]ethyl] ester |
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(OCCOC(=O)C(C)CSCCCCCCCC)=O |
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 |
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. |
Description: |
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. |
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. |
MSDS
COA
LOT NO. |
DOWNLOAD |
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2018-0101 |
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