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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...

詳細記述

保存先:
書誌詳細
出版年:Hum Mol Genet
主要な著者: Yan, Ziying, McCray Jr, Paul B, Engelhardt, John F
フォーマット: Artigo
言語:Inglês
出版事項: Oxford University Press 2019
主題:
オンライン・アクセス: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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