Treatment of Cystic Fibrosis: From Gene- to Cell-Based Therapies
Prognosis of patients with cystic fibrosis (CF) varies extensively despite recent advances in targeted therapies that improve CF transmembrane conductance regulator (CFTR) function. Despite being a multi-organ disease, extensive lung tissue destruction remains the major cause of morbidity and mortal...
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Published in | Frontiers in pharmacology Vol. 12; p. 639475 |
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Main Authors | , , , , |
Format | Journal Article |
Language | English |
Published |
Switzerland
Frontiers Media S.A
16.03.2021
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Subjects | |
Online Access | Get full text |
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Summary: | Prognosis of patients with cystic fibrosis (CF) varies extensively despite recent advances in targeted therapies that improve CF transmembrane conductance regulator (CFTR) function. Despite being a multi-organ disease, extensive lung tissue destruction remains the major cause of morbidity and mortality. Progress towards a curative treatment strategy that implements a
gene addition-technology to the patients' lungs has been slow and not yet developed beyond clinical trials. Improved delivery vectors are needed to overcome the body's defense system and ensure an efficient and consistent clinical response before gene therapy is suitable for clinical care. Cell-based therapy-which relies on functional modification of allogenic or autologous cells
, prior to transplantation into the patient-is now a therapeutic reality for various diseases. For CF, pioneering research has demonstrated proof-of-principle for allogenic transplantation of cultured human airway stem cells into mouse airways. However, applying a cell-based therapy to the human airways has distinct challenges. We review CF gene therapies using viral and non-viral delivery strategies and discuss current advances towards autologous cell-based therapies. Progress towards identification, correction, and expansion of a suitable regenerative cell, as well as refinement of pre-cell transplant lung conditioning protocols is discussed. |
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Bibliography: | ObjectType-Article-2 SourceType-Scholarly Journals-1 ObjectType-Feature-3 content type line 23 ObjectType-Review-1 Edited by: Miquéias Lopes-Pacheco, University of Lisbon, Portugal Massimo Conese, University of Foggia, Italy Reviewed by: Susan Reynolds, Nationwide Children’s Hospital, Columbus, United States This article was submitted to Respiratory Pharmacology, a section of the journal Frontiers in Pharmacology John Sheridan, Cystic Fibrosis Foundation, Bethesda, United States |
ISSN: | 1663-9812 1663-9812 |
DOI: | 10.3389/fphar.2021.639475 |