Gene editing technology for improving life quality: A dream coming true?,Clinical Genetics

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Gene editing technology for improving life quality: A dream coming true?
Clinical Genetics ( IF 2.9 ) Pub Date : 2020-06-07 , DOI: 10.1111/cge.13794
Roya Ramezankhani _{1,

2,

3} , Neda Minaei _{1,

2} , Mahnaz Haddadi ₄ , Shukoofeh Torabi _{1,

2} , Mahdi Hesaraki ₁ , Hamed Mirzaei ₅ , Massoud Vosough _{1,

2} , Catherine M Verfaillie ₆

Affiliation

The fact that monogenic diseases are related to mutations in one specific gene, make gene correction one of the promising strategies in the future to treat genetic diseases or alleviate their symptoms. From this perspective, and along with recent advances in technology, genome editing tools have gained momentum and developed fast. In fact, clustered regularly interspaced short palindromic repeats‐associated protein 9 (CRISPR/Cas9), transcription activator‐like effector nucleases (TALENs), and zinc‐finger nucleases (ZFNs) are regarded as novel technologies which are able to correct a number of genetic aberrations in vitro and in vivo. The number of ongoing clinical trials employing these tools has been increased showing the encouraging outcomes of these tools. However, there are still some major challenges with respect to the safety profile and directed delivery of them. In this paper, we provided updated information regarding the history, nature, methods of delivery, and application of the above‐mentioned gene editing tools along with the meganucleases (an older similar tool) based on published in vitro and in vivo studies and introduced clinical trials which employed these technologies.

中文翻译：

改善生活质量的基因编辑技术：梦想成真？

单基因疾病与一个特定基因的突变有关，这一事实使基因校正成为未来治疗遗传疾病或减轻其症状的有希望的策略之一。从这个角度来看，随着最近技术的进步，基因组编辑工具获得了动力并得到了快速发展。事实上，成簇的规则间隔短回文重复序列相关蛋白 9 (CRISPR/Cas9)、转录激活因子样效应核酸酶 (TALENs) 和锌指核酸酶 (ZFNs) 被认为是能够纠正许多问题的新技术。体外和体内的遗传畸变。使用这些工具的正在进行的临床试验数量有所增加，表明这些工具取得了令人鼓舞的结果。然而，在安全概况和定向交付方面仍然存在一些重大挑战。在本文中，我们根据已发表的体外和体内研究提供了有关上述基因编辑工具以及大范围核酸酶（一种较旧的类似工具）的历史、性质、传递方法和应用的最新信息，并介绍了临床使用这些技术的试验。

更新日期：2020-06-07

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