Key result
NS-065/NCNP-01 strongly promoted exon 53 skipping in a dose-dependent manner and restored dystrophin protein levels in rhabdomyosarcoma and DMD patient-derived cells.
Population
Human rhabdomyosarcoma cell lines and Duchenne muscular dystrophy (DMD) patient-derived cells
Design
Preclinical
Authors
Loading...
Hypothesis-generating for exon 53 skipping in DMD; requires in vivo and clinical validation before any therapeutic consideration.
NS-065/NCNP-01 demonstrates dose-dependent exon 53 skipping and dystrophin restoration in preclinical models, representing a potential therapy for 10.1% of DMD patients.
Watanabe et al. (2018) studied Duchenne muscular dystrophy (DMD). NS-065/NCNP-01 was evaluated on Exon 53 skipping and dystrophin protein restoration. NS-065/NCNP-01 strongly promoted exon 53 skipping in a dose-dependent manner and restored dystrophin protein levels in rhabdomyosarcoma and DMD patient-derived cells.
Synapse has enriched 3 closely related papers on similar clinical questions. Consider them for comparative context: