Optimization of Exon-Skipping Riboswitches and Their Applications to Control Mammalian Cell Fate.

ABSTRACT: Mammalian riboswitches that can regulate transgene expression via RNA-small molecule interaction have promising applications in medicine and biotechnology, as they involve no protein factors that can induce immunogenic reactions and are not dependent on specially engineered promoters. However, the lack of cell-permeable and low-toxicity small molecules and cognate aptamers that can be exploited as riboswitches and the modest switching performance of mammalian riboswitches have limited their applications. In this study, we systematically optimized the design of a riboswitch that regulates exon skipping via an RNA aptamer that binds ASP2905. We examined two design strategies to modulate the stability of the aptamer base stem that blocks the 5' splice site to fine-tune the riboswitch characte

SUBMITTER: Nomura Y 

PROVIDER: S-EPMC11494654 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature