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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Формат:	Online
Язык:	английский
Опубликовано:	MDPI - Multidisciplinary Digital Publishing Institute 2021
Предметы:	cystic fibrosis Staphylococcus aureus superantigen enterotoxin gene cluster MRSA exosomes microvesicles lung primary cells newborn screening trypsinogen CFTR gene next generation sequencing health policy rAAV2/HBoV1 baculovirus insect cells lung microbiome metagenomics gut–lung axis Cystic fibrosis CFTR transcriptomics proteostasis small molecules drug development common and new pathogenic variants ethnic Russian population gene therapy cyclophosphamide transient immunosuppression incidence survival genotype-phenotype correlations health policies CFTR modulators human nasal epithelial cells organoids biomarker functional assay pre-clinical in vitro models CFTR-related disorders molecular diagnosis CFTR variants Next Generation Sequencing (NGS) disease liability interpretation penetrance genotype-guided therapy miRNA airway basal cell lentivirus thema EDItEUR::M Medicine and Nursing
Online-ссылка:	ONIX_20210501_9783039436835_1150
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Molecular Basis and Gene Therapies of Cystic Fibrosis

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