Formulation: A solution in ethanol
Formal Name: 1,1′-[[3-[4-[2-[[3-[bis(2-hydroxyhexadecyl)amino]-2-ethoxypropyl](2-hydroxyhexadecyl)amino]ethyl]-1-piperazinyl]-2-ethoxypropyl]imino]bis-2-hexadecanol
Purity: ≥95%
Formula Markup: C96H197N5O7
Formula Weight: 1533,6
Shelf life (days): 1460
CAS Number: 2639634-93-6
Notes: Lipid C3 is an ionizable cationic lipid (pKa = 5.05-5.671).{49832,68390} It has been used in the formation of lipid nanoparticles (LNPs) for the delivery of mRNA in vitro and in vivo.{49832} Intracerebroventricular administration of LNPs containing lipid C3 and encapsulating mRNA encoding α-L-iduronidase (Idua) to neonates increase midbrain and hippocampal α-L-iduronidase enzyme activity and decrease forebrain, midbrain, and hippocampal glycosaminoglycan levels in an Idua-W392X mouse model of the lysosomal storage disorder mucopolysaccharidosis type I, also known as Hurler syndrome. Intracerebroventricular administration of LNPs containing lipid C3 and encapsulating an mRNA reporter to fetal macaques in utero increase brain GFP expression.
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