RNA modifications in physiology and disease: towards clinical applications
The ability of chemical modifications of single nucleotides to alter the electrostatic charge, hydrophobic surface and base pairing of RNA molecules is exploited for the clinical use of stable artificial RNAs such as mRNA vaccines and synthetic small RNA molecules — to increase or decrease the expre...
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| Veröffentlicht in: | Nature reviews. Genetics 2024-02, Vol.25 (2), p.104-122 |
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| creator | Delaunay, Sylvain Helm, Mark Frye, Michaela |
| description | The ability of chemical modifications of single nucleotides to alter the electrostatic charge, hydrophobic surface and base pairing of RNA molecules is exploited for the clinical use of stable artificial RNAs such as mRNA vaccines and synthetic small RNA molecules — to increase or decrease the expression of therapeutic proteins. Furthermore, naturally occurring biochemical modifications of nucleotides regulate RNA metabolism and function to modulate crucial cellular processes. Studies showing the mechanisms by which RNA modifications regulate basic cell functions in higher organisms have led to greater understanding of how aberrant RNA modification profiles can cause disease in humans. Together, these basic science discoveries have unravelled the molecular and cellular functions of RNA modifications, have provided new prospects for therapeutic manipulation and have led to a range of innovative clinical approaches.
Native nucleotide modifications regulate RNA function and metabolism, the study of which has revealed disease mechanisms, offers therapeutic potential and enables innovative clinical strategies. Chemical modifications in RNA are harnessed for clinical use in stable artificial RNAs such as mRNA vaccines and synthetic small RNA molecules. |
| doi_str_mv | 10.1038/s41576-023-00645-2 |
| format | Article |
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| subjects | 631/208/199 631/208/200 Agriculture Animal Genetics and Genomics Biomedical and Life Sciences Biomedicine Cancer Research Gene Function Human Genetics Hydrophobicity Metabolism mRNA mRNA vaccines Nucleotides Review Article RNA modification |
| title | RNA modifications in physiology and disease: towards clinical applications |
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