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Mini-Review Article

CRISPR系统适用于单个AAV载体传输

卷 22, 期 1, 2022

发表于: 09 December, 2021

页: [1 - 14] 页: 14

弟呕挨: 10.2174/1566523221666211006120355

价格: $65

摘要

CRISPR /Cas基因编辑是一项革命性的技术，可以纠正体内的基因突变，为遗传疾病的治疗干预提供了很大的希望。腺相关病毒(AAV)载体是一种潜在的运载CRISPR/Cas的载体。然而，它们受到其有限的包装容量的限制。识别较小的Cas同源基因，并将其与所需的引导RNA元素一起打包成单个AAV，将是CRISPR/- Cas基因编辑的一个重要优化。扩大Cas蛋白的选择，可以通过单一的AAV交付，不仅增加了翻译应用，也扩大了可用于编辑的基因位点。这篇综述考虑了适用于使用单个AAV载体传递的基因编辑方法的小Cas蛋白同源物的优点和目前的范围。

关键词: CRISPR, CRISPR/Cas9，基因治疗，Cas9，矫形器，AAV。

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图形摘要

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