Adenosine deaminase acting on RNA 1 (ADAR1) (ADAR1)

Adenosine deaminase acting on RNA 1 (ADAR1) is a critical enzyme that catalyzes the hydrolytic deamination of adenosine to inosine (A-to-I) within double-stranded RNA (dsRNA) substrates [UniProt P55265]. This post-transcriptional modification is essential for cellular homeostasis as it marks endogenous dsRNA as self, thereby preventing the inappropriate activation of innate immune sensors like MDA5 and the subsequent induction of a type I interferon response [PubMed: 29466338]. In the biotechnology sector, endogenous ADAR is increasingly utilized as a tool for RNA editing therapeutics, where engineered oligonucleotides recruit the enzyme to specific mRNA sites to correct point mutations or modulate protein function without permanent genomic alteration [Wave Life Sciences; Korro Bio]. Conversely, ADAR1 is a high-priority target for inhibition in oncology; its depletion in cancer cells leads to the accumulation of unedited dsRNA, triggering a viral mimicry response that can overcome resistance to immune checkpoint inhibitors [PubMed: 30651617]. Therapeutic development must carefully balance the precision of RNA editing to avoid transcriptome-wide off-target effects and manage the risk of systemic autoimmunity associated with ADAR1 deficiency [Nature Biotechnology, 2023].

Other names

ADARDSRADIFI4K88DSRBPp110p150Adenosine deaminase acting on RNADouble-stranded RNA-specific adenosine deaminase

Recruitment of endogenous ADAR for site-directed A-to-I RNA editing; Inhibition of ADAR1 catalytic activity to induce viral mimicry and sensitize tumors to immunotherapy.