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Cystic Fibrosis: New Insights into Therapeutic Approaches

Author(s):

Antonella Tosco, Valeria R. Villella, Valeria Raia, Guido Kroemer and Luigi Maiuri*   Pages 1 - 13 ( 13 )

Abstract:


Since the identification of cystic fibrosis (CF) as a disease in 1938 until 2012, only therapies to treat symptoms rather than etiological therapies have been used to treat the disease. Over the last few years new technologies have been developed, and gene editing strategies are now moving toward a one-time cure. This review will summarize recent advances in etiological therapies that target the basic defect in the CF transmembrane receptor (CFTR), the protein that is mutated in CF. We will discuss how newly identified compounds can directly target mutated CFTR to improve its function. Moreover, we will discuss how proteostasis regulators can modify the environment in which mutant CFTR protein is synthesized and decays, thus restoring CFTR function. The future of CF therapies lies in combinatory therapies that may be personalized for each CF patient.

Keywords:

cystic fibrosis, CFTR, modulators, gene editing, repositioning therapy, precision medicine.

Affiliation:

Regional Cystic Fibrosis Center, Pediatric Unit, Department of Translational Medical Sciences, Federico II University, Naples 80131, European Institute for Research in Cystic Fibrosis, Division of Genetics and Cell Biology, San Raffaele Scientific Institute, Milan 20132, Regional Cystic Fibrosis Center, Pediatric Unit, Department of Translational Medical Sciences, Federico II University, Naples 80131, Equipe11 labellisée Ligue Nationale Contrele Cancer, Centre de Recherche des Cordeliers, Paris, European Institute for Research in Cystic Fibrosis, Division of Genetics and Cell Biology, San Raffaele Scientific Institute, Milan 20132



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