CRISPR-Cas系统治疗代谢紊乱的最新方法。

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.2024.07.016
Yajushii Arora, Priya, Manishankar Kumar, Dhruv Kumar
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引用次数: 0

摘要

CRISPR-Cas技术的发展带来了基因组医学的新时代,它为治疗代谢性疾病提供了迄今为止闻所未闻的可能性。CRISPR/ cas9介导的基因治疗中使用的治疗方法强调分布技术,如病毒载体及其在代谢性疾病(如高胆固醇血症、糖原储存病和苯丙酮尿症)的临床前模型中的应用。高通量CRISPR筛选与发现与代谢功能障碍相关的新基因和途径的靶标鉴定的相关性是发现新方法的一个重要方面。CRISPR-Cas技术与干细胞治疗相结合,在基因校正和细胞再生方面具有尖端的选择,为代谢疾病的治疗开辟了新的途径。干细胞治疗与CRISPR-Cas技术的结合是代谢性疾病治疗的重要进展,代谢性疾病由于其复杂的遗传基础而难以治疗。本章介绍了干细胞治疗和CRISPR-Cas系统在治疗多种代谢紊乱方面的最新进展,为有效治疗提供了新的希望。本章研究了最近使用CRISPR-Cas系统解决各种代谢紊乱的技术和发展。本章重点介绍CRISPR-Cas技术的基本工作原理及其在基因编辑、基因敲除和基于激活/抑制的基因修饰方面的潜在用途。
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Current approaches in CRISPR-Cas system for metabolic disorder.

A new era in genomic medicine has been brought by the development of CRISPR-Cas technology, which presents hitherto unheard-of possibilities for the treatment of metabolic illnesses. The treatment approaches used in CRISPR/Cas9-mediated gene therapy, emphasize distribution techniques such as viral vectors and their use in preclinical models of metabolic diseases like hypercholesterolemia, glycogen storage diseases, and phenylketonuria. The relevance of high-throughput CRISPR screens for target identification in discovering new genes and pathways associated with metabolic dysfunctions is an important aspect of the discovery of new approaches. With cutting-edge options for genetic correction and cellular regeneration, the combination of CRISPR-Cas technology with stem cell therapy has opened new avenues for the treatment of metabolic illnesses. The integration of stem cell therapy and CRISPR-Cas technology is an important advance in the treatment of metabolic diseases, which are difficult to treat because of their intricate genetic foundations. This chapter addresses the most recent developments in the application of stem cell therapy and CRISPR-Cas systems to treat a variety of metabolic disorders, providing fresh hope for effective and maybe curative therapies. This chapter examines techniques and developments that have been made recently to address a variety of metabolic disorders using CRISPR-Cas systems. Our chapter focuses on the foundational workings of CRISPR-Cas technology and its potential uses in gene editing, gene knockout, and activation/repression-based gene modification.

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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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