Technologies of gene editing and related clinical trials for the treatment of genetic and acquired diseases: a systematic review

IF 1.2 Q4 GENETICS & HEREDITY Egyptian Journal of Medical Human Genetics Pub Date : 2024-03-07 DOI:10.1186/s43042-024-00501-w
Wessam Sharaf-Eldin
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Abstract

Gene editing can produce irreversible permanent changes to the genetic material at predetermined sequences, avoiding random integration, which is the major drawback of classical gene therapy. The technology has invaded all approaches of genetic engineering and biotechnology with versatile applications in agriculture, industry, and medicine. The present review displays the different approaches and mechanisms of gene editing. Special emphasis has been given to the technology therapeutic applications where all registered clinical trials have been addressed. The Islamic ethical concerns of gene editing have also been highlighted. The great advantages of gene editing technology, coupled with the splendid efforts of scientists to develop systems with superior efficacy and safety would provide an effective avenue for treating a wide range of human diseases in the near future.
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治疗遗传性和获得性疾病的基因编辑技术及相关临床试验:系统综述
基因编辑可以在预定序列上对遗传物质产生不可逆的永久性改变,避免了传统基因疗法的主要缺点--随机整合。该技术已侵入基因工程和生物技术的所有方法,在农业、工业和医学领域有着广泛的应用。本综述介绍了基因编辑的不同方法和机制。其中特别强调了该技术在治疗方面的应用,并对所有已注册的临床试验进行了讨论。此外,还强调了基因编辑的伊斯兰伦理问题。基因编辑技术的巨大优势,加上科学家们为开发具有卓越疗效和安全性的系统所付出的巨大努力,将在不久的将来为治疗各种人类疾病提供有效途径。
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来源期刊
Egyptian Journal of Medical Human Genetics
Egyptian Journal of Medical Human Genetics Medicine-Genetics (clinical)
CiteScore
2.20
自引率
7.70%
发文量
150
审稿时长
18 weeks
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