A Nature Communications study reported that targeted antisense oligonucleotide (ASO) therapy reversed developmental defects in spinal muscular atrophy (SMA) organoid models. The team demonstrated cellular and morphological rescue in human-derived organoids, underscoring ASO strategies’ potential to correct developmental pathologies in neurodegenerative diseases. The paper described molecular mechanisms by which the ASO restored splicing and downstream neuronal maturation. Researchers said the organoid platform enables mechanistic study and preclinical testing of splicing therapeutics. The work strengthens the translational rationale for ASO interventions in SMA and related motor-neuron disorders.