Lipid C3 is an ionizable cationic lipid (pKa = 5.05-5.671).[1],[2] It has been used in the formation of lipid nanoparticles (LNPs) for the delivery of mRNA in vitro and in vivo.[1] 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.
References:
[1].Palanki, R., Bose, S.K., Dave, A., et al.Ionizable lipid nanoparticles for therapeutic base editing of congenital brain disease.ACS Nano17(14)13594-13610(2023).
[2].Billingsley, M.M., Singh, N., Ravikumar, P., et al.Ionizable lipid nanoparticle-mediated mRNA delivery for human car T cell engineeringNano Lett.20(3)1578-1589(2020).