基因编辑技术(GET)可以诱导基因敲低或基因敲除,用于生物医学应用。通过RNAi疗法的基因沉默的临床成功关注其他GET作为治疗方法。这篇评论旨在强调GET,类别,机制,挑战,当前使用,和未来的应用。不同的学术搜索引擎,电子数据库,和选定文章的参考书目被用于本综述的编写,重点是基本考虑因素。目前的结果表明,在GET中,与其他GET相比,CRISPR/Cas9具有更高的编辑效率和靶向特异性,删除,修改,或在宿主基因组中的特定位置替换基因。因此,CRISPR/Cas9在分子生产方面很有才华,组织,cell,和器官疗法。因此,GET可用于发现遗传疾病的创新疗法,大流行,癌症,无望的疾病,器官衰竭。具体来说,GET已被用于生产基因修饰的动物,以避免人体器官衰竭。转基因猪在临床试验中用作心脏来源,肝脏,肾脏,和用于人类异种移植(XT)的肺。病毒,非病毒,和混合载体已被用于递送GET,但有一些限制。因此,细胞外囊泡(EV)被认为是临床应用中GET递送的智能和未来货物。这项研究得出结论,GET有望用于生产分子,细胞,和器官疗法。GET作为XT的使用仍处于早期阶段,它们存在道德和生物安全问题。
Gene editing technologies (GETs) could induce gene knockdown or gene knockout for biomedical applications. The clinical success of gene silence by RNAi therapies pays attention to other GETs as therapeutic approaches. This review aims to highlight GETs, categories, mechanisms, challenges, current use, and prospective applications. The different academic search engines, electronic databases, and bibliographies of selected articles were used in the preparation of this review with a focus on the fundamental considerations. The present results revealed that, among GETs, CRISPR/Cas9 has higher editing efficiency and targeting specificity compared to other GETs to insert, delete, modify, or replace the gene at a specific location in the host genome. Therefore, CRISPR/Cas9 is talented in the production of molecular, tissue, cell, and organ therapies. Consequently, GETs could be used in the discovery of innovative therapeutics for genetic diseases, pandemics, cancer, hopeless diseases, and organ failure. Specifically, GETs have been used to produce gene-modified animals to spare human organ failure. Genetically modified pigs are used in clinical trials as a source of heart, liver, kidneys, and lungs for xenotransplantation (XT) in humans. Viral, non-viral, and hybrid vectors have been utilized for the delivery of GETs with some limitations. Therefore, extracellular vesicles (EVs) are proposed as intelligent and future cargoes for GETs delivery in clinical applications. This study concluded that GETs are promising for the production of molecular, cellular, and organ therapies. The use of GETs as XT is still in the early stage as well and they have ethical and biosafety issues.