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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Publicat a:Nat Commun
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: 2015
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4480796/
https://ncbi.nlm.nih.gov/pubmed/25914116
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ncomms7952
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