| Bioactivity | 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[1][2]. |
| Invitro | TCL053 shows a dissociation constant pKa =6.8[1].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[1]. |
| In Vivo | 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]. |
| Name | TCL053 |
| CAS | 2361162-70-9 |
| Formula | C53H95NO8 |
| Molar Mass | 874.32 |
| Appearance | Liquid |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | Solution, -20°C, 2 years |
| Reference | [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/ |