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

3区 生物学 Q2 Biochemistry, Genetics and Molecular Biology Progress in Molecular Biology and Translational Science Pub Date : 2025-01-01 Epub Date: 2024-08-16 DOI:10.1016/bs.pmbts.2024.07.014
C Bindu Ajaykumar, Sripriya Rajkumar, Bharathi Suresh, Girish Birappa, D A Ayush Gowda, Aparna Jayachandran, Kye-Seong Kim, Seok-Ho Hong, Suresh Ramakrishna
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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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CRISPR/Cas9系统在呼吸系统疾病中的应用进展
遗传和环境因素可对肺部和呼吸系统疾病产生影响,这些疾病与严重症状有关,死亡率高。许多呼吸系统疾病受遗传或表观遗传因素的显著影响。基因治疗为肺部疾病提供了强有力的治疗方法。聚集规律间隔短回文重复序列(CRISPR)和CRISPR相关蛋白9 (CRISPR/Cas9)是一种很有前途的基因修饰工具,可以编辑基因组。利用CRISPR/Cas9系统研究呼吸系统疾病已经取得了一些进展,例如纠正患者来源细胞中的有害突变和改变多种哺乳动物肺部疾病模型中的基因。CRISPR/Cas9研究的进展为肺部疾病的治疗开辟了新的途径。在本章中,我们将讨论几种常见呼吸系统疾病如COPD、哮喘、IPF和ARDS的已知基因和突变。我们进一步回顾了目前在许多呼吸系统疾病和可能的治疗方法中使用CRISPR/Cas9的研究。
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来源期刊
CiteScore
5.00
自引率
0.00%
发文量
110
审稿时长
4-8 weeks
期刊介绍: Progress in Molecular Biology and Translational Science (PMBTS) provides in-depth reviews on topics of exceptional scientific importance. If today you read an Article or Letter in Nature or a Research Article or Report in Science reporting findings of exceptional importance, you likely will find comprehensive coverage of that research area in a future PMBTS volume.
期刊最新文献
Advances in applications of the CRISPR/Cas9 system for respiratory diseases. Advances in CRISPR-Cas systems for kidney diseases. CRISPR challenges in clinical developments. Current approaches in CRISPR-Cas system for metabolic disorder. Current approaches in CRISPR-Cas systems for diabetes.
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