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Open reading frame correction using splice-switching antisense oligonucleotides for the treatment of cystic fibrosis
CFTR gene mutations that result in the introduction of premature termination codons (PTCs) are common in cystic fibrosis (CF). This mutation type causes a severe form of the disease, likely because of low CFTR messenger RNA (mRNA) expression as a result of nonsense-mediated mRNA decay, as well as th...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
National Academy of Sciences
2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8784102/ https://www.ncbi.nlm.nih.gov/pubmed/35017302 http://dx.doi.org/10.1073/pnas.2114886119 |