ssRNAbaserade

ssRNAbaserade is a proposed framework for programmable editing of single-stranded RNA (ssRNA) that would alter individual nucleotides within transcripts without changing the genome. The central idea is to erase or rewrite RNA bases in a targeted, reversible manner, enabling modulation of gene expression at the RNA level.

Mechanism: In concept, ssRNAbaserade would combine an RNA-targeting delivery scaffold—such as an RNA-binding domain or a Cas-derived component adapted for RNA—with a catalytic deaminase or other nucleotide-modifying enzyme. A programmable guide would recruit the editor to a chosen ssRNA site. Edited bases could include A-to-I (read as G) and C-to-U (read as C to T in transcriptional terms), among others, producing a codon or regulatory change in the transcript. Edit windows would depend on the editor design, and edits would persist only while the RNA is present.

Delivery and scope: Editing would be restricted to RNA molecules in the cytoplasm and certain nuclear compartments where ssRNA molecules reside transiently. Speculative delivery methods include lipid nanoparticles or viral vectors, with considerations for tissue specificity and immunogenicity. Because edits occur on RNA, the genome would remain unchanged and effects would be transient, diminishing as RNA molecules turn over.

Applications and limitations: Potential applications include functional genomics studies, transcript-level disease modeling, and temporary correction of pathogenic transcripts. Limitations include off-target edits on non-target RNAs, dependence on target transcript abundance, potential unintended contexts, and challenges in achieving robust efficiency and safety in living systems.

Status: The concept is discussed in theoretical or early-stage discussions; as of now there is no broadly validated experimental demonstration of ssRNAbaserade in vivo.