Development of 2′-O-Methyl and LNA Antisense Oligonucleotides for <i>SMN2</i> Splicing Correction in SMA Cells
Spinal muscular atrophy (SMA) is a devastating neurodegenerative disease caused by mutations in the <i>SMN1</i> gene. Existing therapies demonstrate positive results on SMA patients but still might be ameliorated in efficacy and price. In the presented study we designed antisense oligonu...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2023-11-01
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| Series: | Biomedicines |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2227-9059/11/11/3071 |