Progress of personalized medicine of cystic fibrosis in the times of efficient CFTR modulators

Abstract Background Cystic fibrosis (CF) is a systemic disorder of exocrine glands that is caused by mutations in the CFTR gene. Main body The basic defect in people with CF (pwCF) leads to impaired epithelial transport of chloride and bicarbonate that can be assessed by CFTR biomarkers, i.e. the β-...

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Hlavní autoři:	Burkhard Tümmler, Sophia Theres Pallenberg, Anna-Maria Dittrich, Simon Y. Graeber, Lutz Naehrlich, Olaf Sommerburg, Marcus A. Mall
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	SpringerOpen 2025-05-01
Edice:	Molecular and Cellular Pediatrics
Témata:	CFTR Biomarker Cystic fibrosis Elexacaftor Gene therapy Ivacaftor
On-line přístup:	https://doi.org/10.1186/s40348-025-00194-0
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