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Review
. 2021 Oct 30;19(1):452.
doi: 10.1186/s12967-021-03099-4.

Gene therapy for cystic fibrosis: new tools for precision medicine

Jin-A Lee  1 , Alex Cho #  2 , Elena N Huang #  2 , Yiming Xu  2 , Henry Quach  2 , Jim Hu  2   3 , Amy P Wong  4   5
Affiliations
Review

Gene therapy for cystic fibrosis: new tools for precision medicine

Jin-A Lee et al. J Transl Med. .

Abstract

The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible progress in treating the disease such that the mean survival age of individuals living with CF is now ~58 years in Canada. Recent developments in gene targeting tools and new cell and animal models have re-ignited the search for a permanent genetic cure for all CF. In this review, we highlight some of the more recent gene therapy approaches as well as new models that will provide insight into personalized therapies for CF.

Keywords: Alternative chloride channels; CFTR; Cystic fibrosis; Lung; Organoids; Precision medicine; Stem cells; TMEM16A.

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Conflict of interest statement

Not applicable.

Figures

Fig. 1
Fig. 1
Classes of CFTR variants and CFTR modulators and the impact it has in CFTR expression and processing
Fig. 2
Fig. 2
Graphics of gene editing technologies
Fig. 3
Fig. 3
CF animal models compared to human disease phenotypes
Fig. 4
Fig. 4
Cell models to study CF disease and therapies. For gene editing approaches, green “✓” indicates research data supporting the use of these approaches in the cell models for CF gene correction. Red “X” indicates no information available. For advantages/limitations section, green “✓” indicates possible and red “X” indicates not possible
See this image and copyright information in PMC

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