Targeted Antisense Oligonucleotide Treatment Rescues Developmental Alterations in Spinal Muscular Atrophy Organoids
Irene Faravelli, Paola Rinchetti, Monica Tambalo, Illia Simutin, Lisa Mapelli, Sara Mancinelli, Matteo Miotto, Mafalda Rizzuti, Andrea D’Angelo, Chiara Cordiglieri, Giulia Forotti, Clelia Peano, Paolo Kunderfranco, Luca Calandriello, Giacomo P. Comi, , Stefania Paola Corti
This study shows that early ASO treatment rescues developmental defects in SMA organoids by restoring SMN levels and correcting splicing defects.
Nature Communications · Dec 21, 2025Peer-reviewedNeuroscience