Helmholtz Gemeinschaft


Genome-engineering technologies for modeling and treatment of cystic fibrosis

Item Type:Review
Title:Genome-engineering technologies for modeling and treatment of cystic fibrosis
Creators Name:Dębczyński, M. and Mojsak, D. and Minarowski, Ł. and Maciejewska, M. and Lisowski, P. and Mróz, R.M.
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:Cystic Fibrosis, CRISPR, hiPSC, Prime Editing, Stem Cell-Based Disease Modeling
Source:Advances in Medical Sciences
Page Range:111-120
Date:March 2023
Official Publication:https://doi.org/10.1016/j.advms.2023.02.003
PubMed:View item in PubMed

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