2765519-47-7
Chemical Structure
VL-422
- CAS No.: 2765519-47-7
- Formula:C62H117NO11
- Molecular Weight:1052.59
InChIKey: LUQLMJLIEUBIFP-UHFFFAOYSA-N
SMILES: O=C(OCC(COC(CCCCCCC(OC(CCCCCCCC)CCCCCCCC)=O)=O)COC(CCCCCCC(OC(CCCCCCCC)CCCCCCCC)=O)=O)OCCCN(CC)CC
Biological Activity: VL-422 is an ionizable cationic lipid. VL-422 delivers CRISPR complementary single-guide RNA (sgRNA) and Cas9 mRNA to enable in vitro and in vivo gene editing. LNPs containing VL-422 loaded with Cas9 mRNA and sgRNA targeting the ANGPTL3 gene induce the deletion of premature stop codons within the ANGPTL3 gene in the liver of cynomolgus monkeys. Loss-of-function of ANGPTL3 leads to decreased levels of LDL, HDL and cholesterol in plasma. The VL-422 delivery system can be used for the research of gene editing strategies targeting lipid metabolism diseases[1].
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VL-422 | VL-422 is an ionizable cationic lipid. VL-422 delivers CRISPR complementary single-guide RNA (sgRNA) and Cas9 mRNA to enable in vitro and in vivo gene editing. LNPs containing VL-422 loaded with Cas9 mRNA and sgRNA targeting the ANGPTL3 gene induce the deletion of premature stop codons within the ANGPTL3 gene in the liver of cynomolgus monkeys. Loss-of-function of ANGPTL3 leads to decreased levels of LDL, HDL and cholesterol in plasma. The VL-422 delivery system can be used for the research of gene editing strategies targeting lipid metabolism diseases. | |||||||||||||||||||||
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