Cas9 mRNA (Cap-1; mo⁵U)

Cas9 mRNA (Cap-1; mo⁵U) encodes Cas9, a DNA endonuclease that induces breaks in DNA sequences complementary to single-guide RNA (sgRNA) strands and is commonly used for genome editing.¹ It is capped using a co-transcriptional capping method, resulting in the naturally occurring Cap 1 structure with high capping efficiency. Cas9 mRNA is also polyadenylated and modified with 5-methyoxy-UTP (mo⁵U) to reduce the host cell immune response and enhance mRNA stability. Encapsulation of Cas9 mRNA (Cap-1; mo⁵U) in lipid nanoparticles (LNPs) can be used for mRNA delivery and expression of Cas9 in vitro or in vivo.²

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1. Finn, J.D., Smith, A.R., Patel, M.C., et al. A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing. Cell Rep. 22(9), 2227-2235 (2018).

2. Cui, Y.R., Wang, S.J., Ma, T., et al. KPT330 improves Cas9 precision genome- and base-editing by selectively regulating mRNA nuclear export. Commun. Biol. 5(1), 237 (2022).