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Splicing mutations in the CFTR gene as therapeutic targets
The marketing approval, about ten years ago, of the first disease modulator for patients with cystic fibrosis harboring specific CFTR genotypes (~5% of all patients) brought new hope for their treatment. To date, several therapeutic strategies have been approved and the number of CFTR mutations targ...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Nature Publishing Group UK
2022
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9385490/ https://www.ncbi.nlm.nih.gov/pubmed/35650428 http://dx.doi.org/10.1038/s41434-022-00347-0 |