Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis

Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy... Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent advent of the FDA-approved CFTR modulator drug ivacaftor, alone or in combination with lumacaftor or tezacaftor, has enabled treatment of the majority of patients suffering from CF. Even before the identification of the CFTR gene, gene therapy was put forward as a viable treatment option for this genetic condition. However, initial enthusiasm has been hampered as CFTR gene delivery to the lungs has proven to be more challenging than expected. This review covers the contemporary clinical and scientific knowledge base for small molecule CFTR modulator drug therapy, gene delivery vectors and CRISPR/Cas9 gene editing and highlights the prospect of these technologies for future treatment options. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Gene Therapy Springer Journals

Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis

Gene Therapy, Volume 26 (9) – Jul 12, 2019

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Publisher
Springer Journals
Copyright
Copyright © 2019 by The Author(s), under exclusive licence to Springer Nature Limited
Subject
Biomedicine; Biomedicine, general; Human Genetics; Cell Biology; Nanotechnology; Gene Therapy; Gene Expression
eISSN
1476-5462
DOI
10.1038/s41434-019-0092-5
Publisher site
See Article on Publisher Site

Abstract

Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent advent of the FDA-approved CFTR modulator drug ivacaftor, alone or in combination with lumacaftor or tezacaftor, has enabled treatment of the majority of patients suffering from CF. Even before the identification of the CFTR gene, gene therapy was put forward as a viable treatment option for this genetic condition. However, initial enthusiasm has been hampered as CFTR gene delivery to the lungs has proven to be more challenging than expected. This review covers the contemporary clinical and scientific knowledge base for small molecule CFTR modulator drug therapy, gene delivery vectors and CRISPR/Cas9 gene editing and highlights the prospect of these technologies for future treatment options.

Journal

Gene TherapySpringer Journals

Published: Jul 12, 2019

References

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