Highly sensitive screening of antisense sequences for different types of DMD mutations in patients' urine-derived cells
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DOI: 10.1016/j.jns.2021.117337
Abstract: Exon skipping using short antisense oligonucleotides (AONs) is a promising treatment for Duchenne muscular dystrophy (DMD). Several exon-skipping drugs, including viltolarsen (NS-065/NCNP-01), have been approved worldwide. Immortalized human skeletal muscle cell lines, such as rhabdomyosarcoma… read more here.
Keywords: rhabdomyosarcoma cells; highly sensitive; derived cells; exon skipping ... See more keywords