DG9-Conjugated Morpholino-Based Exon 51-Skipping Therapy for Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the DMD gene that leads to the loss of the dystrophin protein. Exon-skipping therapy with phosphorodiamidate morpholino oligomers (PMOs) has been shown to restore the disrupted...

Thrilled to share our new Methods in Molecular Biology chapter. link.springer.com/protocol/10....　We describe in vivo methods for systemic DG9-PMO delivery, exon 51 skipping, and efficacy assessment in hDMDdel52;mdx mice. #DMD #RNAtherapeutics #ASO #DG9 #ExonSkipping @SpringerNature