dCas13-eNAT10

dCas13-eNAT10 is a programmable RNA epigenetic editing platform designed for site-specific RNA acetylation. It consists of a catalytically inactive Cas13b (dCas13b) protein fused to an engineered, hyperactive variant of the human N4-acetylcytidine (ac4C) acetyltransferase NAT10 (eNAT10). The eNAT10 component is a truncated version of the native enzyme, optimized for higher catalytic activity and lacking the nucleolar localization signal to ensure efficient targeting of specific transcripts in the cytoplasm and nucleus. By employing guide RNAs (gRNAs), the system precisely 'writes' ac4C modifications on targeted mRNAs, which has been shown to increase protein translation efficiency and promote the cytoplasmic enrichment of the modified transcripts. Developed by researchers at the Korea Advanced Institute of Science and Technology (KAIST), the system has been successfully delivered in vivo using a split-intein dual-AAV8 vector system to target endogenous Angptl3 in mouse liver, demonstrating its potential as a therapeutic modality for diseases associated with ac4C dysregulation, such as cancers, neurological disorders, and metabolic conditions.

Other names

dCas13b-eNAT10dCas-13b-eNAT10dCas 13b-eNAT10programmable RNA acetylation platform