C14-490
C14-490 is an ionizable cationic lipid used for the formulation of lipid nanoparticles (LNPs) for mRNA delivery. C14-490 LNPs can be surface-functionalized with CD45 antibodies to enable targeted delivery to hematopoietic stem cells (HSCs), mediate efficient mRNA delivery and CRISPR-Cas9 gene editing in fetal HSCs in utero, with potent and durable genome modulation in HSCs and their progeny. C14-490 is a key component of the Systematically optimized Targeted Editing Machinery (STEM) LNPs for in vivo HSC gene editing applications.
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- CAS. Nr.: 2639634-82-3
- Formel: C86H177N5O7
- Molecular Weight:1393.35
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Speicherung:
Solution, -20°C, 2 years
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Biologische Aktivität
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Chemical Information
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Help & FAQs
Biologische Aktivität
C14-490 LNPs CD45 antibody conjugation to C14-490 LNPs increases average particle diameter by ~20 nm without altering PDI or mRNA encapsulation efficiency[1].
C14-490 LNPs conjugated to CD45 antibody (100 ng total mRNA, 24 h incubation) boost GFP mRNA delivery efficiency eightfold in Jurkat cells; 24 h administration of 100 ng mRNA leads to 35% GFP+ cells with no decline in cell viability[1].
C14-490 LNPs conjugated to CD45 antibody (25-150 ng total mRNA; 24 h) exhibit dose-dependent improvement in mRNA delivery to Jurkat cells, with maximum 10-fold enhancement at 50 ng mRNA per 30,000 cells after 24 h incubation[1].
C14-490 LNPs conjugated to CD45 antibody (100 ng total mRNA for Jurkat cells, 25 ng total mRNA for HepG2 cells; 24 h transfection, 30 min antibody pre-incubation) have enhanced mRNA delivery efficacy that is CD45 receptor-specific, as pre-treatment with free CD45 antibodies blocks the effect, and no enhancement is observed in CD45-negative HepG2 cells[1].
C14-490 ionizable lipid formulated into LNPs exhibit consistent size, low polydispersity, and high encapsulation efficiency[1].
C14-490 ionizable lipid in LNPs correlates with enhanced relative GFP knockout efficacy in HepG2-GFP cells, with a positive correlation coefficient of +0.34[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
14C-490 LNPs adopting optimized B5 formulation (1 mg/kg mRNA; i.v.; single in utero injection at E13.5) achieve higher intrauterine TTR locus gene editing efficiency in fetal liver than the unmodified A0 formulation[1].
14C-490 LNPs adopting optimized B5 formulation (1 mg/kg mRNA, i.v., single in utero injection at E13.5) yield fourfold enhanced TTR locus gene editing in fetal mouse HSCs compared with non-targeted B5-formulated counterparts post in utero injection[1]
C14-490 LNPs (1 mg/kg; in utero i.v.; single dose) drive robust GFP expression in fetal mouse hepatocytes, with minimal expression in fetal hematopoietic stem cells[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:R26mT/mG (B6.129(Cg)-Gt(ROSA)26Sortm4(ACTB-tdTomato,-EGFP)Luo/J) mice (fetal, gestational day 13.5)[1]
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Dosage:1 mg/kg mRNA
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Administration:i.v.; single in utero injection at E13.5
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Result:Mediated 50% transfection of fetal hepatocytes and 2% transfection of fetal HSCs (Lin-/Sca1+/cKit+).
Mediated 50% transfection of adult hepatocytes and no detectable genome modulation in adult bone marrow HSCs.
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Animal Model:BALB/c mice (fetal, gestational day 13.5)[1]
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Dosage:1 mg/kg total mRNA
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Administration:i.v.; single in utero injection at E13.5
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Result:Mediated threefold greater indels at the TTR locus in fetal liver tissue compared to unoptimized A0 C14-490 LNPs.\nMediated ~6% indels at the TTR locus in whole fetal liver tissue for both STEM and untargeted B5 formulations.
Mediated ~8% indels in fetal HSCs, which was fourfold higher than the ~2% indels mediated by untargeted B5 LNPs in fetal HSCs.
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Animal Model:R26mT/mG (E13.5 fetuses, in utero model)[1]
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Dosage:1 mg/kg
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Administration:in utero i.v.; single dose
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Result:Achieved a mean GFP+ percentage of 53% in fetal hepatocytes (CD45-/CD31-).
Showed non-significant mean GFP+ percentage in fetal hematopoietic stem cells (Lin-/Sca1+/cKit+).
Displayed widespread GFP fluorescence across whole fetuses and dissected fetal liver tissue via stereomicroscopy.
Chemical Information
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CAS. Nr. 2639634-82-3
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Appearance Liquid
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Molecular Weight 1393.35
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Formel C86H177N5O7
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Color Colorless to light yellow
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SMILES
OC(CCCCCCCCCCCC)CN(CC(CN(CC(CCCCCCCCCCCC)O)CC(CCCCCCCCCCCC)O)OCC)CCN1CCN(CC(CN(CC(CCCCCCCCCCCC)O)CC(CCCCCCCCCCCC)O)OCC)CC1
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Versand
Room temperature in continental US; may vary elsewhere.
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Speicherung
Solution, -20°C, 2 years
Reinheit & Dokumentation
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Data Sheet (271 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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Handling Instructions (2659 KB)
Verweise
Calculators
Konzentration (Stammlösung) × Volumen (Stammlösung) = Konzentration (Ziellösung) × Volumen (Ziellösung)
C14-490 Related Classifications
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