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Gene editing using large insertions

delete2026-09-17
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Alexandra Despang *
DOI:10.1038/s41587-026-03319-6delete
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摘要

摘要

En 中文
尽管碱基编辑等小规模基因编辑技术已进入临床试验,但大片段基因载体的应用面临不同挑战,包括编辑效率低、递送载体尺寸限制和潜在的免疫原性。利用重组酶、转座酶(包括CRISPR相关转座酶CASTs)、基因组写入系统和反转座子等可编程基因组工程系统为治疗提供了不同途径,但每种系统均有独特局限性。如何进一步开发这些技术最具前景的策略是什么?哪些参数需要改进以实现高效且安全的治疗?我们邀请了该领域三位专家分享他们对挑战与机遇的看法。

期刊

Nature Biotechnology 封面图
Nature Biotechnology
IF:
41.7
论文数:
1.2W
被引数:
10.1W

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