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Steric Inhibition of 5′ UTR Regulatory Elements Results in Upregulation of Human CFTR
Cystic fibrosis (CF) is an autosomal recessive monogenic disease caused by mutations in the CFTR gene. Therapeutic approaches that are focused on correcting CFTR protein face the challenge of the heterogeneity in CFTR mutations and resulting defects. Thus, while several small molecules directed at C...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2019
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6822282/ https://www.ncbi.nlm.nih.gov/pubmed/31351782 http://dx.doi.org/10.1016/j.ymthe.2019.06.016 |