Correction of Airway Stem Cells: Genome Editing Approaches for the Treatment of Cystic Fibrosis

Cystic fibrosis (CF) is an autosomal recessive disease caused by variations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Although CF affects multiple organs, the primary cause of mortality is respiratory failure resulting from poor clearance of hyperviscous secretions and...

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Vydáno v:Hum Gene Ther
Hlavní autoři: King, Nicholas E., Suzuki, Shingo, Barillà, Cristina, Hawkins, Finn J., Randell, Scott H., Reynolds, Susan D., Stripp, Barry R., Davis, Brian R.
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc., publishers 2020
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7495916/
https://ncbi.nlm.nih.gov/pubmed/32741223
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2020.160
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