Programmable RNA editing with endogenous ADAR enzymes - a feasible option for the treatment of inherited retinal disease?

ABSTRACT: RNA editing holds great promise for the therapeutic correction of pathogenic, single nucleotide variants (SNV) in the human transcriptome since it does not risk creating permanent off-targets edits in the genome and has the potential for innovative delivery options. Adenine deaminases acting on RNA (ADAR) enzymes catalyse the most widespread form of posttranscriptional RNA editing in humans and their ability to hydrolytically deaminate adenosine to inosine in double stranded RNA (dsRNA) has been harnessed to change pathogenic single nucleotide variants (SNVs) in the human genome on a transcriptional level. Until now, the most promising target editing rates have been achieved by exogenous delivery of the catalytically active ADAR deaminase domain (ADAR_DD) fused to an RNA binding

SUBMITTER: Bellingrath JS 

PROVIDER: S-EPMC10244592 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature