Shelf life (days): 730
Info: An ionizable cationic lipid (pKa = 6.3); LNPs containing TCL065 and encapsulating sgRNA targeting the donor site of dystrophin exon 45, in combination with TCL065-containing LNPs encapsulating sgRNA targeting the splicing acceptor site of dystrophin exon 45, restore dystrophin expression in myoblasts differentiated from iPS cells isolated from patients with DMD; LNPs containing TCL065 and encapsulating mRNA encoding the Cas9 nuclease, in combination with LNPs containing TCL065 and encapsulating single-guide RNA targeting the Rosa26 locus, have been used to induce CRISPR-mediated gene editing in the mouse gastrocnemius muscle
Formulation: A solution in methyl acetate
Formal Name: 2-(((5-(dimethylamino)pentanoyl)oxy)methyl)-2-(((3-pentyloctanoyl)oxy)methyl)propane-1,3-diyl bis(3-pentyloctanoate)
Purity: ≥95%
Formula Markup: C51H97NO8
Formula Weight: 852.31782
CAS Number: 1872269-57-2
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