Clinical progress in genome-editing technology and in vivo delivery techniques.
Clinical progress in genome-editing technology and in vivo delivery techniques.
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DOI：
10.1016/j.tig.2022.12.001
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发表时间：
2023-01
期刊：
影响因子：
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通讯作者：
Jennifer Khirallah;Maximilan Eimbinder;Yamin Li;Qiaobing Xu
中科院分区：
文献类型：
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作者：
Jennifer Khirallah;Maximilan Eimbinder;Yamin Li;Qiaobing Xu
人们对应用基因组编辑工具来预防、治疗和治愈各种疾病产生了广泛的兴趣。自从发现成簇规则间隔短回文重复 (CRISPR)/CRISPR 相关蛋白 (Cas) 系统以来，这些技术已与不同的递送系统结合使用，以创建高效的治疗方案。每种输送系统都有其自身的优点和缺点，并且用于各种应用。随着大量基因编辑应用正在研究中，但很少被引入临床，我们回顾了该领域的当前进展，特别是基因组编辑在体内和临床中的应用，并确定了当前的挑战和未来增长的领域。
There is wide interest in applying genome-editing tools to prevent, treat, and cure a variety of diseases. Since the discovery of the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein (Cas) systems, these techniques have been used in combination with different delivery systems to create highly efficacious treatment options. Each delivery system has its own advantages and disadvantages and is being used for various applications. With the large number of gene-editing applications being studied but very few being brought into the clinic, we review current progress in the field, specifically where genome editing has been appliedin vivoand in the clinic, and identify current challenges and areas of future growth.