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TCL053 is an ionizable lipid carrier and used to introduce active components, in particular nucleic acids, into cells with excellent efriciency. TCL053, together with DPPC (Dipalmitoylphosphatidylcholine), PEG-DMG (Polyethylene glycoldimyristoyl glycerol), and cholesterol, forms lipid nanoparticle (LNP) which is able to deliver Cas9 mRNA and sgRNA into skeletal muscle.

TCL053 shows a dissociation constant pKa =6.8. TCL053 : DPPC : Cholesterol : DMG-PEG =60 : 10.6 : 27.3 : 2.1, shows an encapsulation rate of 96%, and a size of 79.1 nM.

TCL053-based LNP encapsulating Cas9 mRNA (TCL053-LNP-CRISPR) (20 µg total RNA; i.m.) exhibits high genome editing and exon skipping efficacy, and is higher than other in vivo mRNA delivery reagent (in vivo-jetRNA).
TCL053-based LNP, via limb perfusion method, can target multiple muscle groups, with repeated administration and low immunogenicity features.
TCL053-based LNP acts as a delivery vehicle of CRISPR-Cas9 and can be used for skeletal muscle disorders research.
TCL053-based LNP induces stable genomic exon skipping and restore dystrophin protein in a DMD mouse model that harbors a humanized exon sequence.

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