Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders
Abstract Antisense oligonucleotides (ASOs) are incredibly versatile molecules that can be designed to specifically target and modify RNA transcripts to slow down or halt rare genetic disease progression. They offer the potential to target groups of patients or can be tailored for individual cases. N...
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| Автори: | , , , |
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| Формат: | Artigo |
| Мова: | Inglês |
| Опубліковано: |
Nature Portfolio
2024-01-01
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| Серія: | Communications Medicine |
| Онлайн доступ: | https://doi.org/10.1038/s43856-023-00419-1 |
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