Formulation: A solution in ethanol
Formal Name: 4-(dimethylamino)-butanoic acid, (10Z,13Z)-1-(9Z,12Z)-9,12-octadecadien-1-yl-10,13-nonadecadien-1-yl ester
Purity: ≥95%
Formula Markup: C43H79NO2
Formula Weight: 642,1
Shelf life (days): 730
CAS Number: 1224606-06-7
Notes: DLin-MC3-DMA is an ionizable cationic lipid (apparent pKa = 6.44) that has been used in the generation of lipid nanoparticles (LNPs) encapsulating siRNA, mRNA, or plasmid DNA for use in vitro and in vivo.{54974,60923,66839} LNPs containing DLin-MC3-DMA accumulate in the muscle and non-draining lymph nodes after intramuscular administration and in the spleen after intravenous administration in mice.{66839} DLin-MC3-DMA-containing LNPs encapsulating siRNA targeting F7, the gene encoding Factor VII, reduce hepatic and plasma Factor VII levels in mice without increasing serum levels of alanine transaminase (ALT), aspartate aminotransferase (AST), or bile acids, which are all markers of liver toxicity.{68850} Formulations containing DLin-MC3-DMA have been used in LNPs encapsulating transthyretin-directed siRNA for the treatment of hereditary transthyretin-mediated amyloidosis-induced polyneuropathy.
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