Therapeutic Nonsense Suppression Modalities: From Small Molecules to Nucleic Acid-Based Approaches
Nonsense mutations are genetic mutations that create premature termination codons (PTCs), leading to truncated, defective proteins in diseases such as cystic fibrosis, neurofibromatosis type 1, Dravet syndrome, Hurler syndrome, Beta thalassemia, inherited bone marrow failure syndromes, Duchenne musc...
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| Principais autores: | , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
MDPI AG
2024-06-01
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| Serier: | Biomedicines |
| Fag: | |
| Online adgang: | https://www.mdpi.com/2227-9059/12/6/1284 |
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