TCL053 是一种可电离的脂质，用于高效地将活性成分 (特别是核酸) 导入细胞。TCL053 能够与二棕榈酰磷脂酰胆碱 (DPPC)、聚乙二醇二肉豆蔻酰基甘油 (PEG-DMG) 和胆固醇，一起形成脂质纳米粒 (LNP)，能够将 Cas9 mRNA 和 sgRNA 传递到骨骼肌中。

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.

Liquid

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. has not independently confirmed the accuracy of these methods. They are for reference only.

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. has not independently confirmed the accuracy of these methods. They are for reference only.

Room temperature in continental US; may vary elsewhere.

Solution, -20°C, 2 years

[1]. Kenjo E, et al. Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice. Nat Commun. 2021 Dec 8;12(1):7101.
[2]. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8654819/