Formulation: A solution in methyl acetate
Formal Name: 9Z,12Z-octadecadienoic acid, 3-[4,4-bis(octyloxy)-1-oxobutoxy]-2-[[[[3-(diethylamino)propoxy]carbonyl]oxy]methyl]propyl ester
Purity: ≥95%
Formula Markup: C50H93NO9
Formula Weight: 852,3
Shelf life (days): 730
CAS Number: 1799316-64-5
Notes: LP-01 is an ionizable cationic lipid (pKa = ~6.1).{71100} It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of single-guide RNA (sgRNA) and mRNA in vitro and in vivo.{71100,49897} LNPs containing LP-01 and encapsulating both Cas9 mRNA and modified sgRNA for the transport protein transthyretin (Ttr) induce gene editing in liver cells in mice in a dose-dependent manner resulting in reduced serum Ttr levels for at least 12 months.{71100} Subretinal administration of LNPs containing LP-01 and encapsulating both Cas9 mRNA and sgRNA targeting the loxP stop codon induce retinal gene editing in Ai9 mice.{49897}
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