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...

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Detalles Bibliográficos
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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2015
Materias:
Article
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

Internet

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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