CRISPR-Cas系统治疗肾脏疾病的进展

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: 2024-08-17 DOI:10.1016/bs.pmbts.2024.07.020
Bhupendra Puri, Yogesh A Kulkarni, Anil Bhanudas Gaikwad
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引用次数: 0

摘要

CRISPR-Cas系统的最新进展彻底改变了肾脏疾病的研究和治疗,包括急性肾损伤(AKI)、慢性肾病(CKD)、糖尿病肾病(DKD)、狼疮肾炎(LN)和多囊肾病(PKD)。CRISPR-Cas技术为单基因肾病(如PKD和Alport综合征)的遗传修饰提供了精确和通用的工具。CRISPR技术的最新进展也显示出治疗其他肾脏疾病如AKI、CKD和DKD的希望。CRISPR-Cas有望编辑这些疾病背后的基因突变,可能导致更有效、更持久的治疗。此外,CRISPR-Cas系统的适应性允许开发定制的治疗策略,专门针对导致不同肾脏疾病的遗传和分子机制。除了DNA修饰,CRISPR-Cas技术还可以编辑肾脏疾病中的非编码RNA,如lncrna和mirna。尽管取得了这些进展,但仍然存在重大挑战,包括特定肾细胞的递送效率和潜在的脱靶效应。然而,CRISPR-Cas技术的快速发展对未来肾脏疾病的管理产生了变革性的影响,通过个性化和精确的治疗方法提供了提高患者预后的潜力。本章重点介绍了CRISPR-Cas系统的最新进展及其在各种肾脏疾病中的潜在应用。
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Advances in CRISPR-Cas systems for kidney diseases.

Recent advances in CRISPR-Cas systems have revolutionised the study and treatment of kidney diseases, including acute kidney injury (AKI), chronic kidney disease (CKD), diabetic kidney disease (DKD), lupus nephritis (LN), and polycystic kidney disease (PKD). CRISPR-Cas technology offers precise and versatile tools for genetic modification in monogenic kidney disorders such as PKD and Alport syndrome. Recent advances in CRISPR technology have also shown promise in addressing other kidney diseases like AKI, CKD, and DKD. CRISPR-Cas holds promise to edit genetic mutations underlying these conditions, potentially leading to more effective and long-lasting treatments. Furthermore, the adaptability of CRISPR-Cas systems allows for developing tailored therapeutic strategies that specifically target the genetic and molecular mechanisms contributing to different kidney diseases. Beyond DNA modifications, CRISPR-Cas technologies also enable editing noncoding RNA, such as lncRNAs and miRNAs, in kidney diseases. Despite these advancements, significant challenges persist, including delivery efficiency to specific kidney cells and potential off-target effects. However, the rapid progress in CRISPR-Cas technology suggests a transformative impact on the future management of kidney diseases, offering the potential for enhanced patient outcomes through personalised and precise therapeutic approaches. This chapter highlights the recent advancement of CRISPR-Cas systems and their potential applications in various kidney diseases.

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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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