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Seamlessly bridge your LNP discovery journey from RUO to bulk GLP-tox and GMP excipient-grade material.
Explore CGMP Lipid ServicesLP-01 is an ionizable cationic lipid (pKa = ~6.1).1 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.1,2 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.1 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.2
WARNING This product is not for human or veterinary use.
1. A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing. Cell Rep. 22(9), 2227-2235 (2018).
2. Preformed vesicle approach to LNP manufacturing enhances retinal mRNA delivery. Small e2400815 (2024).