Advances in applications of the CRISPR/Cas9 system for respiratory diseases.

Prog Mol Biol Transl Sci

Graduate School of Biomedical Science and Engineering, Hanyang University, Seoul, Korea; College of Medicine, Hanyang University, Seoul, Korea. Electronic address:

Published: January 2025

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Article Abstract

Genetic and environmental factors can have an impact on lung and respiratory disorders which are associated with severe symptoms and have high mortality rates. Many respiratory diseases are significantly influenced by genetic or epigenetic factors. Gene therapy offers a powerful approach providing therapeutic treatment for lung diseases. Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein 9 (CRISPR/Cas9) are promising gene modifying tool that can edit the genome. The utilization of CRISPR/Cas9 systems in the investigation of respiratory disorders has resulted in advancements such as the rectification of deleterious mutations in patient-derived cells and the alteration of genes in multiple mammalian lung disease models. New avenues of treatment for lung disorders have been opened up by advances in CRISPR/Cas9 research. In this chapter, we discuss the known genes and mutations that cause several common respiratory disorders such as COPD, asthma, IPF, and ARDS. We further review the current research using CRISPR/Cas9 in numerous respiratory disorders and possible therapeutic treatments.

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http://dx.doi.org/10.1016/bs.pmbts.2024.07.014DOI Listing

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