[基因编辑正在改变遗传性疾病的治疗方法]。

Lakartidningen Pub Date : 2025-01-29
C I Edvard Smith, Rula Zain, Pontus Blomberg
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引用次数: 0

摘要

基因编辑是基因治疗领域的一项新技术,它可以精确地改变染色体DNA的序列。即使有替代策略,获得诺贝尔奖的CRISPR/Cas技术也已成为主导原则。近年来,碱基编辑和引物编辑技术得到了发展,它们允许在没有DNA双链断裂的情况下进行编辑。第一个临床基因编辑结果于2021年报告;从那时起,许多患者得到了治疗,最近第一个治疗方法在英国被批准为一种新疗法,后来在美国和欧盟。此更新描述了包括方法学发展和安全性在内的各个方面。
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[Gene editing is changing the treatment of hereditary diseases].

Gene editing is a novel technology within gene therapy, which changes sequences in chromosomal DNA with precision. Even if there are alternative strategies, the Nobel Prize-winning CRISPR/Cas technology has become the dominating principle. During recent years base editing and prime editing, permitting editing without DNA double-strand breaks, have been developed. The first clinical gene editing results were reported in 2021; since then many patients have been treated, and recently the first treatment was approved as a novel therapy in the UK and later in USA and in EU. This update describes various aspects including methodological developments and safety.

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来源期刊
Lakartidningen
Lakartidningen Medicine-Medicine (all)
CiteScore
0.30
自引率
0.00%
发文量
134
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