Applications of CRISPR technologies to the development of gene and cell therapy.

IF 2.9 3区 生物学 Q3 BIOCHEMISTRY & MOLECULAR BIOLOGY
Chul-Sung Park, Omer Habib, Younsu Lee, J. Hur
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引用次数: 0

Abstract

Advancements in gene and cell therapy have resulted in novel therapeutics for diseases previously considered incurable or challenging to treat. Among the various contributing technologies, genome editing stands out as one of the most crucial for the progress in gene and cell therapy. The discovery of CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) and the subsequent evolution of genetic engineering technology have markedly expanded the field of target-specific gene editing. Originally studied in the immune systems of bacteria and archaea, the CRISPR system has demonstrated wide applicability to effective genome editing of various biological systems including human cells. The development of CRISPR-based base editing has enabled directional cytosine-tothymine and adenine-to-guanine substitutions of select DNA bases at the target locus. Subsequent advances in prime editing further elevated the flexibility of the edit multiple consecutive bases to desired sequences. The recent CRISPR technologies also have been actively utilized for the development of in vivo and ex vivo gene and cell therapies. We anticipate that the medical applications of CRISPR will rapidly progress to provide unprecedented possibilities to develop novel therapeutics towards various diseases.
将 CRISPR 技术应用于基因和细胞疗法的开发。
基因和细胞疗法的进步为以前被认为无法治愈或难以治疗的疾病带来了新的疗法。在各种促进技术中,基因组编辑技术是基因和细胞疗法取得进展的最关键技术之一。CRISPR(Clustered Regularly Interspaced Short Palindromic Repeats)的发现以及随后基因工程技术的发展,极大地拓展了靶向基因编辑领域。CRISPR 系统最初是在细菌和古细菌的免疫系统中进行研究的,现已广泛应用于包括人类细胞在内的各种生物系统的有效基因组编辑。基于 CRISPR 的碱基编辑技术的发展实现了目标基因座上特定 DNA 碱基的胞嘧啶-胸腺嘧啶和腺嘌呤-鸟嘌呤定向替换。随后的素材编辑技术进一步提高了将多个连续碱基编辑为所需序列的灵活性。最近,CRISPR 技术还被积极用于体内和体外基因和细胞疗法的开发。我们预计,CRISPR 的医学应用将迅速发展,为开发针对各种疾病的新型疗法提供前所未有的可能性。
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
BMB Reports
BMB Reports 生物-生化与分子生物学
CiteScore
5.10
自引率
7.90%
发文量
141
审稿时长
1 months
期刊介绍: The BMB Reports (BMB Rep, established in 1968) is published at the end of every month by Korean Society for Biochemistry and Molecular Biology. Copyright is reserved by the Society. The journal publishes short articles and mini reviews. We expect that the BMB Reports will deliver the new scientific findings and knowledge to our readers in fast and timely manner.
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