Document Type
Article
Publication Date
6-6-2023
Publication Title
Cells
Abstract
Cystic fibrosis (CF) is a monogenic recessive genetic disorder caused by mutations in the CF Transmembrane-conductance Regulator gene (CFTR). Remarkable progress in basic research has led to the discovery of highly effective CFTR modulators. Now ~90% of CF patients are treatable. However, these modulator therapies are not curative and do not cover the full spectrum of CFTR mutations. Thus, there is a continued need to develop a complete and durable therapy that can treat all CF patients once and for all. As CF is a genetic disease, the ultimate therapy would be in-situ repair of the genetic lesions in the genome. Within the past few years, new technologies, such as CRISPR/Cas gene editing, have emerged as an appealing platform to revise the genome, ushering in a new era of genetic therapy. This review provided an update on this rapidly evolving field and the status of adapting the technology for CF therapy.
PubMed ID
37371025
Volume
12
Issue
12
Creative Commons License
This work is licensed under a Creative Commons Attribution 4.0 International License.
Recommended Citation
Wang, Guoshun, "Genome Editing for Cystic Fibrosis" (2023). School of Medicine Faculty Publications. 1278.
https://digitalscholar.lsuhsc.edu/som_facpubs/1278
10.3390/cells12121555
Included in
Amino Acids, Peptides, and Proteins Commons, Congenital, Hereditary, and Neonatal Diseases and Abnormalities Commons, Medical Genetics Commons