Molecular Basis and Gene Therapies of Cystic Fibrosis

Summary of Genes. Thirty years ago, the gene responsible for cystic fibrosis (CF), a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator gene, was identified. This progress has considerably changed our understanding of the pathophysiology of CF an...

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Bibliographic Details
Format:	eBook
Language:	English
Published:	Basel, Switzerland MDPI - Multidisciplinary Digital Publishing Institute 2020
Subjects:	Medicine airway basal cell baculovirus biomarker CFTR CFTR gene CFTR modulators CFTR variants CFTR-related disorders common and new pathogenic variants cyclophosphamide cystic fibrosis Cystic fibrosis disease liability drug development enterotoxin gene cluster ethnic Russian population exosomes functional assay gene therapy genotype-guided therapy genotype-phenotype correlations gut-lung axis health policies health policy human nasal epithelial cells incidence insect cells interpretation lentivirus lung lung microbiome metagenomics microvesicles miRNA molecular diagnosis MRSA newborn screening next generation sequencing Next Generation Sequencing (NGS) organoids penetrance pre-clinical in vitro models primary cells proteostasis rAAV2/HBoV1 small molecules Staphylococcus aureus superantigen survival transcriptomics transient immunosuppression trypsinogen
Online Access:	Open Access: DOAB: description of the publication Open Access: DOAB, download the publication

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Open Access: DOAB: description of the publication
Open Access: DOAB, download the publication

Molecular Basis and Gene Therapies of Cystic Fibrosis

Internet

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