Immortalized canine dystrophic myoblast cell lines for development of peptide-conjugated splice-switching oligonucleotides
Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease caused by frameshift or nonsense mutations in the <i>DMD</i> gene, resulting in the loss of dystrophin from muscle membranes. Exon skipping using splice-switching oligonucleotides (SSOs) restores the reading frame of &l...
Main Authors: | , , , , , , , , , , , , , , |
---|---|
Format: | Journal article |
Language: | English |
Published: |
Mary Ann Liebert
2021
|