Ziclumeran

cas9 mRNA cas9 mRNA (mRNA000042) messenger RNA (mRNA), 5' capped, encoding codon-optimised clustered regularly interspaced short palindromic repeats (CRISPR) associated protein 9 (Cas9), flanked by untranslated regions (UTR) with significant sequence homology to the Xenopus beta globin gene, and terminated with a polyadenylation (polyA) tail. A Kozak sequence precedes the open reading frame and the 5' cap is a synthetic anti-reverse cap analogue (ARCA); contains N1-methylpseudouridine instead of uridine (all-U>m1Ψ) NTLA-2001 component mRNA000042 RNA (recombinant 5′-(m7Gm-(5′→5′)-ppp-G)-capped all uridine→N1-methylpseudouridine-substituted synthetic CRISPR-associated endodeoxyribonuclease CAS9 low codon A-optimized-specifying plus Xenopus laevis β-globin 5′-and 3′-untranslated flanking region-containing poly(A)-tailed messenger) ziclumeran [INN] ZICLUMERAN [USAN]