Formulation: A solution in methyl acetate
Formal Name: 2-(((4-(dimethylamino)butanoyl)oxy)methyl)-2-((((Z)-tetradec-9-enoyl)oxy)methyl)propane-1,3-diyl (9Z,9'Z)-bis(tetradec-9-enoate)
Purity: ≥95%
Formula Markup: C53H95NO8
Formula Weight: 874,3
Shelf life (days): 365
CAS Number: 2361162-70-9
Notes: TCL053 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.{66116} 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.
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