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Article

Gene therapy for cystic fibrosis: new tools for precision medicine.

Journal of translational medicine - 30 Oct 2021

Lee Jin-A, Cho Alex, Huang Elena N, Xu Yiming, Quach Henry, Hu Jim, Wong Amy P

Abstract excerpt

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...

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Identifiers

PMID: 34717671
DOI: 10.1186/s12967-021-03099-4
PMCID: PMC8556969

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