Researchers reported in Nature Communications that targeted antisense oligonucleotide (ASO) therapy reversed developmental defects in spinal muscular atrophy (SMA) organoid models. The study showed ASO-mediated modulation of SMN splicing restored cellular phenotypes and improved motor neuron development in human-derived organoids. The work provides a preclinical bridge between genetic mechanism and therapeutic intervention, supporting ASO strategies beyond systemic delivery and into organoid-based screening. Biotech developers pursuing SMA and other splice-modulating therapies may use these organoid results to refine dosing, delivery approaches and biomarker strategies ahead of clinical translation.