C14-O2

C14-O2 is an ionizable cationic lipid (apparent pK_a = 6.6) that has been used in the generation of lipid nanoparticles (LNPs) for the delivery of mRNA, single-guide RNA (sgRNA), and protein in vitro and in vivo.^1,2 LNPs containing C14-O2 and encapsulating mRNA encoding a chimeric antigen receptor (CAR) induce CAR expression and B cell aplasia in mice. LNPs containing C14-O2 and encapsulating mRNA encoding the Cas9 nuclease and sgRNA targeting the gene encoding transthyretin (TTR) induce TTR insertion and deletion (indel) formation in the lungs without inducing TTR indel formation in the liver or reducing TTR levels in the serum in mice.³ C14-O2-containing LNPs encapsulating Cas9 protein induce a higher amount of gene editing in mouse lungs than those encapsulating mRNA encoding Cas9.² LNPs containing C14-O2 and encapsulating Cas9 protein also induce chloride ion influx to a greater degree than those encapsulating Cas9 mRNA in human-derived bronchial epithelial gene-edited (16HBEge) cells containing the ΔF508 cystic fibrosis transmembrane conductance regulator (CFTR) mutation.

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1. Mukalel, A.J., Hamilton, A.G., Billingsley, M.M., et al. Oxidized mRNA lipid nanoparticles for in situ chimericantigen receptor monocyte engineering. Adv. Funct. Mater. 34(27), 2312038 (2024).

2. Joseph, R.A., Haley, R.M., Padilla, M.S., et al. Cas9 protein outperforms mRNA in lipid nanoparticle-mediated CFTR repair. Nano Lett. (2025).

3. Haley, R.M., Padilla, M.S., El-Mayta, R.D., et al. Lipid nanoparticles for in vivo lung delivery of CRISPR-Cas9 ribonucleoproteins allow gene editing of clinical targets. ACS Nano 19(14), 13790-13804 (2025).