Delivering the CRISPR/Cas9 system for engineering gene therapies: Recent cargo and delivery approaches for clinical translation.
Delivering the CRISPR/Cas9 system for engineering gene therapies: Recent cargo and delivery approaches for clinical translation.
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为基因工程疗法提供CRISPR/Cas9系统：用于临床翻译的最新货物和交付方法。
DOI：
10.3389/fbioe.2022.973326
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发表时间：
2022
影响因子：
5.7
通讯作者：
Jonas, Steven J.
中科院分区：
文献类型：
--
作者：
Foley, Ruth A.;Sims, Ruby A.;Duggan, Emily C.;Olmedo, Jessica K.;Ma, Rachel;Jonas, Steven J.
簇状规则间隔短回文重复相关蛋白9(CRISPR/Cas9)改变了我们选择性编辑人类基因组的能力。这项技术已经迅速成为最标准化和可重复性最好的基因编辑工具。CRISPR/Cas9系统促进了生物医学研究和基因工程的快速发展，为预防和治疗目前无法治愈的单基因和更复杂的人类疾病提供了巨大的潜力。然而，CRISPR/CAS9的临床应用仍然存在重大障碍。虽然在体外、体外和体内的基因编辑已经在实验室环境中得到了广泛的证明，但目前转化为临床研究的方法受到将CRISPR/Cas9相关试剂输送到预期治疗靶点的精确度、可扩展性和效率方面的不足。为了克服这些挑战，最近的进展同时操纵了运送货物和运输CRISPR/CAS9试剂的车辆。在选择通知送货车辆的货物时，必须对两者的精度和效率进行优化。本文综述了目前应用CRISPR/Cas9基因编辑工具开发新兴细胞疗法的生物工程方法，重点介绍了它的两个主要可工程化组件：运送载体及其携带的基因编辑载体。在CRISPR/CAS9为广泛的临床翻译进行优化的关键考虑的背景下，讨论了当代生物医学应用的障碍。
Clustered Regularly Interspaced Short Palindromic Repeats associated protein 9 (CRISPR/Cas9) has transformed our ability to edit the human genome selectively. This technology has quickly become the most standardized and reproducible gene editing tool available. Catalyzing rapid advances in biomedical research and genetic engineering, the CRISPR/Cas9 system offers great potential to provide diagnostic and therapeutic options for the prevention and treatment of currently incurable single-gene and more complex human diseases. However, significant barriers to the clinical application of CRISPR/Cas9 remain. While in vitro, ex vivo, and in vivo gene editing has been demonstrated extensively in a laboratory setting, the translation to clinical studies is currently limited by shortfalls in the precision, scalability, and efficiency of delivering CRISPR/Cas9-associated reagents to their intended therapeutic targets. To overcome these challenges, recent advancements manipulate both the delivery cargo and vehicles used to transport CRISPR/Cas9 reagents. With the choice of cargo informing the delivery vehicle, both must be optimized for precision and efficiency. This review aims to summarize current bioengineering approaches to applying CRISPR/Cas9 gene editing tools towards the development of emerging cellular therapeutics, focusing on its two main engineerable components: the delivery vehicle and the gene editing cargo it carries. The contemporary barriers to biomedical applications are discussed within the context of key considerations to be made in the optimization of CRISPR/Cas9 for widespread clinical translation.
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影响因子：
10.2
作者：
Ankrett DN;Carugo D;Lei J;Glynne-Jones P;Townsend PA;Zhang X;Hill M
通讯作者：
Hill M
影响因子：
15.1
作者：
Chou, Shih-Jie;Yang, Peng;Chiou, Shih-Hwa
通讯作者：
Chiou, Shih-Hwa
DOI：
10.1073/pnas.1917125117
发表时间：
2020-05-19
影响因子：
11.1
作者：
Belling, Jason N.;Heidenreich, Liv K.;Jonas, Steven J.
通讯作者：
Jonas, Steven J.
影响因子：
3.2
作者：
Carugo, Dario;Ankrett, Dyan N.;Hill, Martyn
通讯作者：
Hill, Martyn
DOI：
10.1002/smll.202100546
发表时间：
2021-07
期刊：
Small (Weinheim an der Bergstrasse, Germany)
影响因子：
--
作者：
Ban Q;Yang P;Chou SJ;Qiao L;Xia H;Xue J;Wang F;Xu X;Sun N;Zhang RY;Zhang C;Lee A;Liu W;Lin TY;Ko YL;Antovski P;Zhang X;Chiou SH;Lee CF;Hui W;Liu D;Jonas SJ;Weiss PS;Tseng HR
通讯作者：
Tseng HR