For research use only. Not for therapeutic Use.
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].
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].
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].
| Catalog Number | I043377 |
| CAS Number | 2361162-70-9 |
| Synonyms | [2-[4-(dimethylamino)butanoyloxymethyl]-3-[(Z)-tetradec-9-enoyl]oxy-2-[[(Z)-tetradec-9-enoyl]oxymethyl]propyl] (Z)-tetradec-9-enoate |
| Molecular Formula | C53H95NO8 |
| Purity | ≥95% |
| InChI | InChI=1S/C53H95NO8/c1-6-9-12-15-18-21-24-27-30-33-36-40-49(55)59-45-53(48-62-52(58)43-39-44-54(4)5,46-60-50(56)41-37-34-31-28-25-22-19-16-13-10-7-2)47-61-51(57)42-38-35-32-29-26-23-20-17-14-11-8-3/h15-20H,6-14,21-48H2,1-5H3/b18-15-,19-16-,20-17- |
| InChIKey | MJPUTLHISFHJJZ-QMJZYSMNSA-N |
| SMILES | CCCCC=CCCCCCCCC(=O)OCC(COC(=O)CCCCCCCC=CCCCC)(COC(=O)CCCCCCCC=CCCCC)COC(=O)CCCN(C)C |
| 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/ |
