From cell culture to non-human primates: navigating preclinical models for RNA therapeutics

“...predicting whether a mutation, intronic or exonic, actually causes a splicing defect? That is something AI tools are genuinely good at...”Jokūbas Leikauskas, Editor, Nucleic Acid Insights , speaks with Jin Rong Ow, Scientist, A*STAR Institute of Molecular and Cell Biology, and Tommaso Tabaglio, Senior Scientist, A*STAR Institute of Molecular and Cell Biology, about selecting and improving preclinical models for RNA therapeutic development, covering the strengths and limitations of different experimental systems, off-target assessment, chemistry-driven selectivity, and the future of oligonucleotide design workflows.