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Expert Opin Ther Targets. 2018 Feb;22(2):177-189. doi: 10.1080/14728222.2018.1406922. Epub 2017 Dec 13.

TGFβ as a therapeutic target in cystic fibrosis.

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a Department of Pediatrics , Cincinnati Children's Hospital Medical Center , Cincinnati , OH , USA.


Cystic fibrosis (CF) is a genetic disease characterized by progressive lung disease. Most CF therapies focus on treating secondary pulmonary complications rather than addressing the underlying processes inducing airway remodeling and ineffective response to infection. Transforming growth factor beta (TGFβ) is a cytokine involved in fibrosis, inflammation, and injury response as well as a genetic modifier and biomarker of CF lung disease. Targeting the TGFβ pathway has been pursued in other diseases, but the mechanism of TGFβ effects in CF is less well understood. Areas covered: In this review, we discuss CF lung disease pathogenesis with a focus on potential links to TGFβ. TGFβ signaling in lung health and disease is reviewed. Recent studies investigating TGFβ's impact in CF airway epithelial cells are highlighted. Finally, an overview of potential therapies to target TGFβ signaling relevant to CF are addressed. Expert opinion: The broad impact of TGFβ signaling on numerous cellular processes in homeostasis and disease is both a strength and a challenge to developing TGFβ dependent therapeutics in CF. We discuss the challenges inherent in developing TGFβ-targeted therapy, identifying appropriate patient populations, and questions regarding the timing of treatment. Future directions for research into TGFβ focused therapeutics are discussed.


Cystic fibrosis; cystic fibrosis transmembrane conductance regulator; genetic modifier; transforming growth factor beta

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