Optimizing In Vitro Efficacy Assessment of the Antisense Oligonucleotide Nusinersen in Human Cellular Models
<b>Background/Objectives:</b> Spinal muscular atrophy (SMA) is a rare genetic disorder caused by mutations or deletions in <i>SMN1</i>, resulting in the loss of SMN protein and severe neuromuscular consequences. Nusinersen, an antisense oligonucleotide that promotes full-length SMN2 transcript forma...
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| Auteurs principaux: | , , , |
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| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
MDPI AG
2026-05-01
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| Collection: | Pharmaceutics |
| Sujets: | |
| Accès en ligne: | https://www.mdpi.com/1999-4923/18/6/652 |
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