Non-target RNA transcript

Non-target RNA transcripts refer to the collective population of RNA molecules within a cell that are not the intended focus of a therapeutic intervention but are inadvertently affected by it. This phenomenon is a significant concern for RNA-targeted modalities, including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and CRISPR-based systems like Cas13, which can bind to or cleave these transcripts due to partial sequence complementarity or non-specific interactions [1, 2]. Such unintended interactions can lead to the degradation of essential cellular RNAs, resulting in off-target toxicity, cellular dormancy, or other adverse clinical outcomes [2]. In drug development, the identification and minimization of effects on non-target RNA transcripts are critical for ensuring the specificity and safety of the therapeutic candidate. Advanced bioinformatics and high-throughput sequencing technologies are typically employed to predict and monitor these off-target effects during the preclinical and clinical phases of drug discovery [1].

Other names

Off-target RNANon-specific RNA transcriptBystander RNA

Unintended hybridization, cleavage, or degradation by RNA-targeting therapeutic agents due to sequence complementarity or non-specific binding.