A Nature Communications study shows that targeted antisense oligonucleotide (ASO) therapy reverses developmental defects in spinal muscular atrophy (SMA) organoid models. Investigators reported restoration of molecular and cellular hallmarks of normal development after ASO treatment, offering evidence that early molecular correction can rescue neurodevelopmental trajectories in human‑derived tissues. The work supports the clinical rationale for early ASO intervention and adds organoid‑based phenotypic rescue as a translational bridge between genetic insights and therapeutic testing. Antisense therapy here functions as a precision tool to modulate RNA splicing and protein expression in disease models.