Researchers reported in Nature Communications that a targeted antisense oligonucleotide (ASO) therapy reversed developmental defects in spinal muscular atrophy (SMA) organoid models. The study showed morphological and molecular rescue in human-derived organoids after ASO treatment, demonstrating a potential route to correct developmental pathology in vitro. The results provide a translational bridge from molecular correction to tissue‑level repair and support continued ASO development for SMA. ASOs are short synthetic nucleic acids that modulate RNA splicing or expression; this study highlights their capacity to restore developmental programs in human models. The work will influence preclinical pipelines and may inform dose and biomarker strategies for next‑generation SMA trials.