Peptide-conjugated PMOs for the treatment of myotonic dystrophy
Antisense oligonucleotides (ASOs) have shown great therapeutic potential in the treatment of many neuromuscular diseases including myotonic dystrophy 1 (DM1). However, systemically delivered ASOs display poor biodistribution and display limited penetration into skeletal muscle. The conjugation of ce...
| Главные авторы: | , , , , , |
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| Другие авторы: | |
| Формат: | Journal article |
| Язык: | English |
| Опубликовано: |
Springer
2022
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