Potential of Cell-Penetrating Peptide-Conjugated Antisense Oligonucleotides for the Treatment of SMA
Spinal muscular atrophy (SMA) is a severe neuromuscular disorder that is caused by mutations in the survival motor neuron 1 (<i>SMN1</i>) gene, hindering the production of functional survival motor neuron (SMN) proteins. Antisense oligonucleotides (ASOs), a versatile DNA-like drug, are a...
| Hoofdauteurs: | , |
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| Formaat: | Artikel |
| Taal: | English |
| Gepubliceerd in: |
MDPI AG
2024-06-01
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| Reeks: | Molecules |
| Onderwerpen: | |
| Online toegang: | https://www.mdpi.com/1420-3049/29/11/2658 |