Molecular BiologybioRxivPreprint
Mdx mice dosed with antisense to CD49d and dystrophin exon skip morpholino; reduced force loss and affected gene expression pathways support ATL1102 combination therapy in DMD patients
Advait Suchet Padhye, Leonit Kiriaev, Chrystal F. Tiong…·George Tachas
This study shows that combining ASO targeting CD49d with PMO exon skipping improves muscle function and modulates gene expression in mdx mice, supporting ATL1102 combination therapy for DMD.
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