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...
| 主要な著者: | , , , , , , , , , , , , , , |
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| フォーマット: | Journal article |
| 言語: | English |
| 出版事項: |
Mary Ann Liebert
2021
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