Advances in CRISPR/Cas systems-based cell and gene therapy.

3区 生物学 Q2 Biochemistry, Genetics and Molecular Biology
Arpita Poddar, Farah Ahmady, Prashanth Prithviraj, Rodney B Luwor, Ravi Shukla, Shakil Ahmed Polash, Haiyan Li, Suresh Ramakrishna, George Kannourakis, Aparna Jayachandran
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引用次数: 0

Abstract

Cell and gene therapy are innovative biomedical strategies aimed at addressing diseases at their genetic origins. CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) systems have become a groundbreaking tool in cell and gene therapy, offering unprecedented precision and versatility in genome editing. This chapter explores the role of CRISPR in gene editing, tracing its historical development and discussing biomolecular formats such as plasmid, RNA, and protein-based approaches. Next, we discuss CRISPR delivery methods, including viral and non-viral vectors, followed by examining the various engineered CRISPR variants for their potential in gene therapy. Finally, we outline emerging clinical applications, highlighting the advancements in CRISPR for breakthrough medical treatments.

基于 CRISPR/Cas 系统的细胞和基因疗法的进展。
细胞和基因治疗是一种创新的生物医学策略,旨在从基因源头解决疾病问题。CRISPR(Clustered Regularly Interspaced Short Palindromic Repeats)系统已成为细胞和基因治疗的开创性工具,为基因组编辑提供了前所未有的精确性和多功能性。本章将探讨 CRISPR 在基因编辑中的作用,追溯其历史发展,并讨论生物分子格式,如基于质粒、RNA 和蛋白质的方法。接下来,我们讨论了 CRISPR 的传递方法,包括病毒载体和非病毒载体,然后研究了各种 CRISPR 工程变体在基因治疗中的潜力。最后,我们概述了新兴的临床应用,重点介绍了 CRISPR 在突破性医学治疗方面取得的进展。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
6.90
自引率
0.00%
发文量
0
审稿时长
>12 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.
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