Science and Research

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

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

  • Tümmler, B.
  • Pallenberg, S. T.
  • Dittrich, A. M.
  • Graeber, S. Y.
  • Naehrlich, L.
  • Sommerburg, O.
  • Mall, M. A.

Keywords

  • Biomarker
  • Cftr
  • Cystic fibrosis
  • Elexacaftor
  • Gene therapy
  • Ivacaftor
  • Tezacaftor
  • Theratyping
Publication details
DOI: 10.1186/s40348-025-00194-0
Journal: Mol Cell Pediatr
Pages: 6 
Number: 1
Work Type: Review
Location: Assoziierter Partner, BREATH, TLRC, UGMLC
Disease Area: CFBE
Partner / Member: BIH, JLU, MHH, UKHD
Access-Number: 40320452


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