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Correction of F508del CFTR in airway epithelium using nanoparticles delivering triplex-forming PNAs
Cystic fibrosis (CF) is a lethal genetic disorder most commonly caused by the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is not readily amenable to gene therapy because of its systemic nature and challenges including in vivo gene delivery and transien...
Autores principales: | McNeer, Nicole Ali, Anandalingam, Kavitha, Fields, Rachel J., Caputo, Christina, Kopic, Sascha, Gupta, Anisha, Quijano, Elias, Polikoff, Lee, Kong, Yong, Bahal, Raman, Geibel, John P, Glazer, Peter M., Saltzman, W. Mark, Egan, Marie E. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4480796/ https://www.ncbi.nlm.nih.gov/pubmed/25914116 http://dx.doi.org/10.1038/ncomms7952 |
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