Between hope and reality: treatment of genetic diseases through nucleic acid-based drugs
Abstract Rare diseases (RD) affect a small number of people compared to the general population and are mostly genetic in origin. The first clinical signs often appear at birth or in childhood, and patients endure high levels of pain and progressive loss of autonomy frequently associated with short l...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Portfolio
2024-04-01
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| Series: | Communications Biology |
| Online Access: | https://doi.org/10.1038/s42003-024-06121-9 |