Stereo-random oligonucleotides enable efficient recruitment of ADAR in vitro and in vivo.

ABSTRACT: Site-directed RNA editing is a promising and potentially safer alternative to genome editing. Previous methods have been developed that recruit the endogenously and ubiquitously expressed ADAR enzymes to initiate site-specific A-to-I edits, but often suffer from low efficacy or dependency on viral delivery. Chemically modified oligonucleotides may be a promising alternative, but the approach still lacks systematic in-depth studies. Furthermore, the best characterized platform uses stereo-pure backbone chemistry, which is not widely used, commercially unavailable and challenging to manufacture. Here, we report on single-stranded oligonucleotides of 30-60 nt length, which are fully chemically stabilized by applying commercially available, classical RNA drug modifications, like 2´-O-methyl, 2

SUBMITTER: Pfeiffer LS 

PROVIDER: S-EPMC12494997 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature