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Seamlessly bridge your LNP discovery journey from RUO to bulk GLP-tox and GMP excipient-grade material.
Explore CGMP Lipid ServicesTCL053 is an ionizable cationic lipid (pKa = 6.8) that has been used in the generation of lipid nanoparticles (LNPs) for the delivery of mRNA and single-guide RNA (sgRNA) in vitro and in vivo.1 LNPs containing TCL053 and encapsulating sgRNA targeting the donor site of dystrophin exon 45, in combination with TCL053-containing LNPs encapsulating sgRNA targeting the splicing acceptor site of dystrophin exon 45, restore dystrophin expression in myoblasts differentiated from induced-pluripotent stem (iPS) cells isolated from patients with Duchenne muscular dystrophy (DMD). LNPs containing TCL053 and encapsulating mRNA encoding the Cas9 nuclease, in combination with LNPs containing TCL053 and encapsulating single-guide RNA targeting the Rosa26 locus, have been used to induce CRISPR-mediated gene editing in the mouse gastrocnemius muscle.
WARNING This product is not for human or veterinary use.
1. Low immunogenicity of LNP allows repeated administrations of CRISPR-