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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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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Yan, Ziying, McCray Jr, Paul B, Engelhardt, John F
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2019
Assuntos:
Acesso em linha: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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