Article ; Online: Current Treatment Options for Cystic Fibrosis-Related Liver Disease.
International journal of molecular sciences
2020 Volume 21, Issue 22
Abstract: Cystic Fibrosis-related liver disease (CFLD) has become a leading cause of morbidity and mortality in patients with Cystic Fibrosis (CF), and affects children and adults. The understanding of the pathogenesis of CFLD is key in order to develop ... ...
Abstract | Cystic Fibrosis-related liver disease (CFLD) has become a leading cause of morbidity and mortality in patients with Cystic Fibrosis (CF), and affects children and adults. The understanding of the pathogenesis of CFLD is key in order to develop efficacious treatments. However, it remains complex, and has not been clarified to the last. The search for a drug might be additionally complicated due to the diverse clinical picture and lack of a unified definition of CFLD. Although ursodeoxycholic acid has been used for decades, its efficacy in CFLD is controversial, and the potential of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators and targeted gene therapy in CFLD needs to be defined in the near future. This review focuses on the current knowledge on treatment strategies for CFLD based on pathomechanistic viewpoints. |
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MeSH term(s) | Cystic Fibrosis/complications ; Cystic Fibrosis/genetics ; Cystic Fibrosis/metabolism ; Cystic Fibrosis/therapy ; Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Cystic Fibrosis Transmembrane Conductance Regulator/metabolism ; Humans ; Liver Diseases/etiology ; Liver Diseases/genetics ; Liver Diseases/metabolism ; Liver Diseases/pathology ; Ursodeoxycholic Acid/therapeutic use |
Chemical Substances | CFTR protein, human ; Cystic Fibrosis Transmembrane Conductance Regulator (126880-72-6) ; Ursodeoxycholic Acid (724L30Y2QR) |
Language | English |
Publishing date | 2020-11-14 |
Publishing country | Switzerland |
Document type | Journal Article ; Review |
ZDB-ID | 2019364-6 |
ISSN | 1422-0067 ; 1422-0067 ; 1661-6596 |
ISSN (online) | 1422-0067 |
ISSN | 1422-0067 ; 1661-6596 |
DOI | 10.3390/ijms21228586 |
Database | MEDical Literature Analysis and Retrieval System OnLINE |
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