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Expert Opin Biol Ther. 2018 Mar;18(3):281-292. doi: 10.1080/14712598.2018.1413087. Epub 2017 Dec 8.

The long and winding road: stem cells for cystic fibrosis.

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a Laboratory of Experimental and Regenerative Medicine, Department of Medical and Surgical Sciences , University of Foggia , Foggia , Italy.
b Department of Medicine and Health Sciences 'V. Tiberio' , University of Molise , Campobasso , Italy.
c Cystic Fibrosis Center, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Department of Pathophysiology and Transplantation , University of Milan , Milan , Italy.
d Division of Internal Medicine and Chronobiology Unit , IRCCS 'Casa Sollievo della Sofferenza' , San Giovanni Rotondo (FG) , Italy.



Cystic fibrosis (CF) is a genetic syndrome with a high mortality rate due to severe lung disease. Despite having several drugs targeting specific mutated CFTR proteins already in clinical trials, new therapies, based on stem cells, are also emerging to treat those patients.


The authors review the main sources of stem cells, including embryonic stem cells (ESCs), induced-pluripotent stem cells (iPSCs), gestational stem cells, and adult stem cells, such as mesenchymal stem cells (MSCs) in the context of CF. Furthermore, they describe the main animal and human models of lung physiology and pathology, involved in the optimization of these stem cell-applied therapies in CF.


ESCs and iPSCs are emerging sources for disease modeling and drug discovery purposes. The allogeneic transplant of healthy MSCs, that acts independently to specific mutations, is under intense scrutiny due to their secretory, immunomodulatory, anti-inflammatory and anti-bacterial properties. The main challenge for future developments will be to get exogenous stem cells into the appropriate lung location, where they can regenerate endogenous stem cells and act as inflammatory modulators. The clinical application of stem cells for the treatment of CF certainly warrants further insight into pre-clinical models, including large animals, organoids, decellularized organs and lung bioengineering.


Cystic fibrosis transmembrane conductance regulator; bioengineered lungs; decellularized lungs; embryonic stem cells; induced pluripotent stem cells; mesenchymal stem cells; organoids

[Indexed for MEDLINE]

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