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...
| Autori principali: | , , , , , |
|---|---|
| Altri autori: | |
| Natura: | Journal article |
| Lingua: | English |
| Pubblicazione: |
Springer
2022
|