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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Publicado en:Nat Commun
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: Artigo
Lenguaje:Inglês
Publicado: 2015
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Article
Acceso en línea: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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