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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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में बचाया:

ग्रंथसूची विवरण
अन्य लेखक:	Engelhardt, John (संपादक), Ferec, Claude (संपादक), Yan, Ziying (संपादक)
स्वरूप:	इलेक्ट्रोनिक पुस्तक अध्याय
भाषा:	अंग्रेज़ी
प्रकाशित:	Basel, Switzerland MDPI - Multidisciplinary Digital Publishing Institute 2020
विषय:	Medicine 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
ऑनलाइन पहुंच:	DOAB: download the publication DOAB: description of the publication
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