Cell-penetrating peptide-conjugated Morpholino rescues SMA in a symptomatic preclinical model
Margherita Bersani, Mafalda Rizzuti, Elisa Pagliari, Manuela Garbellini, Domenica Saccomanno, Hong M. Moulton, Nereo Bresolin, Giacomo P. Comi, Stefania Corti, Monica Nizzardo
Topics & Concepts
Spinal muscular atrophySMA*MorpholinoMedicineDiseaseBioinformaticsPharmacologyCancer researchBiologyPathologyZebrafishGeneBiochemistryCombinatoricsMathematicsNeurogenetic and Muscular Disorders ResearchRNA modifications and cancerCongenital Anomalies and Fetal Surgery