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Review
. 2023 Mar;68(1):111-120.
doi: 10.1016/j.advms.2023.02.003. Epub 2023 Mar 12.

Genome-engineering technologies for modeling and treatment of cystic fibrosis

Michał Dębczyński  1 , Damian Mojsak  2 , Łukasz Minarowski  2 , Monika Maciejewska  2 , Paweł Lisowski  3 , Robert M Mróz  2
Affiliations
Review

Genome-engineering technologies for modeling and treatment of cystic fibrosis

Michał Dębczyński et al. Adv Med Sci. 2023 Mar.

Erratum in

Abstract

Cystic fibrosis (CF) is an autosomal recessive disease caused by defects in the CF transmembrane conductance regulator (CFTR) protein. Due to the genetic nature of the disease, interventions in the genome can target any underlying alterations and potentially provide permanent disease resolution. The current development of gene-editing tools, such as designer nuclease technology capable of genome correction, holds great promise for both CF and other genetic diseases. In recent years, Cas9-based technologies have enabled the generation of genetically defined human stem cell and disease models based on induced pluripotent stem cells (iPSC). In this article, we outline the potential and possibilities of using CRISPR/Cas9-based gene-editing technology in CF modeling.

Keywords: CRISPR; Cystic fibrosis; Prime editing; Stem cell-based disease modeling; hiPSC.

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Conflict of interest statement

Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

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