2361162-70-9

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)^[1].
TCL053-based LNP, via limb perfusion method, can target multiple muscle groups, with repeated administration and low immunogenicity features^[1].
TCL053-based LNP acts as a delivery vehicle of CRISPR-Cas9 and can be used for skeletal muscle disorders research^[1].
TCL053-based LNP induces stable genomic exon skipping and restore dystrophin protein in a DMD mouse model that harbors a humanized exon sequence^[1].