EcRESCUE: a novel ecDHFR-regulated RESCUE system with reduced RNA off-targeting activity.

ABSTRACT: The currently available RESCUE RNA base editing system demonstrates considerable potential for the treatment of genetic diseases at the transcriptional level. However, the relatively high incidence of off-target events hampers the precise RNA editing, thereby limiting its use in the clinical setting. This study describes a new RNA base editing method, named ecRESCUE, which utilizes inducible stabilization of the protein ecDHFR DD fused at the C-terminal of the original RESCUE system. In vitro experiments in 293T cells showed that the ecRESCUE editor markedly reduced the incidence of off-target single nucleotide polymorphisms without affecting the RNA A-to-I and C-to-U base editing efficiency. Altogether, these results demonstrate that the inducible ecRESCUE system represents an attractive

SUBMITTER: Wang Y 

PROVIDER: S-EPMC8325194 | biostudies-literature | 2021 Jul

REPOSITORIES: biostudies-literature