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针对基因疗法的最小免疫原核酶进行理性工程

Rumya Raghavan1,2,3,4,5,6, Mirco J Friedrich1,2,3,4,5, Indigo King7

  • 1Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.

Nature communications
|January 3, 2025
PubMed
概括

工程CRISPR-Cas基因编辑工具显示免疫性降低,用于更安全的遗传疾病治疗. 这些修改后的核酶保持高活性,同时逃避免疫检测,为改进的临床应用铺平了道路.

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