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...
محفوظ في:
| المؤلفون الرئيسيون: | , , , |
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| التنسيق: | Artigo |
| اللغة: | Inglês |
| منشور في: |
Nature Portfolio
2024-01-01
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| سلاسل: | Communications Medicine |
| الوصول للمادة أونلاين: | https://doi.org/10.1038/s43856-023-00419-1 |
| الوسوم: |
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