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Advances in gene therapy for cystic fibrosis lung disease

Cystic fibrosis (CF) is a multiorgan recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Gene therapy efforts have focused on treating the lung, since it manifests the most significant life-threatening disease. Over two decades have p...

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Bibliographic Details
Published in:Hum Mol Genet
Main Authors: Yan, Ziying, McCray Jr, Paul B, Engelhardt, John F
Format: Artigo
Language:Inglês
Published: Oxford University Press 2019
Subjects:
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6796993/
https://ncbi.nlm.nih.gov/pubmed/31332440
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddz139
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