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What Can RNA-Based Therapy Do for Monogenic Diseases?
The use of RNA-based approaches to treat monogenic diseases (i.e., hereditary disorders caused by mutations in single genes) has been developed on different fronts. One approach uses small antisense oligonucleotides (ASOs) to modulate RNA processing at various stages; namely, to enhance correct spli...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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MDPI
2023
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9863139/ https://www.ncbi.nlm.nih.gov/pubmed/36678889 http://dx.doi.org/10.3390/pharmaceutics15010260 |