000 01275nam a2200121Ia 4500
008 220620s9999||||xx |||||||||||||| ||und||
245 0 _aMolecular Basis and Gene Therapies of Cystic Fibrosis
546 _aEnglish[eng]
650 _acystic 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
700 _aEngelhardt, John||Ferec, Claude||Yan, Ziying
856 _uhttps://mdpi.com/books/pdfview/book/3205
942 _cEB
999 _c34598
_d34598