Description
An ionizable cationic lipid (pKa = 6.8); 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 iPS cells isolated from patients with 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