The long and winding road: Stem cells for cystic fibrosis

Massimo Conese, Elisa Beccia, Stefano Castellani, Sante di Gioia, Carla Colombo, Antonella Angiolillo, Annalucia Carbone

Research output: Contribution to journalReview articlepeer-review

Abstract

Introduction: 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. Areas covered: 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 cellapplied therapies in CF. Expert opinion: 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.

Original languageEnglish
Pages (from-to)281-292
JournalExpert Opinion on Biological Therapy
Volume18
Issue number3
DOIs
Publication statusPublished - Jan 1 2018

Keywords

  • Bioengineered lungs
  • Cystic fibrosis transmembrane conductance regulator
  • Decellularized lungs
  • Embryonic stem cells
  • Induced pluripotent stem cells
  • Mesenchymal stem cells
  • Organoids

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry

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