Effect of NS-065/NCNP-01 in Cells Derived from Patients with DMD
(A) Schedule of cell differentiation, PMO transfection, RT-PCR, and western blot. (B) Exon 53 skipping induced by NS-065/NCNP-01 in cells derived from patients with deletion of either exons 45–52 (n = 4) or exons 48–52 (n = 3). Each point represents the mean ± SD. (C) Dystrophin protein expression in cells derived from a patient with DMD involving deletion of exons 48–52 transfected with NS-065/NCNP-01 at final concentrations of up to 10 μmol/L. DMD, Duchenne muscular dystrophy; PMO, phosphorodiamidate morpholino oligomer.