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基因纠正调节性T细胞疗法用于IL2RA缺乏症

Alvin S Ha1, Nechama Kalter2, Michael Rosenberg2

  • 1Department of Laboratory Medicine, University of California, San Francisco, San Francisco, CA 94143, USA; Gladstone-UCSF Institute of Genomic Immunology, San Francisco, CA 94158, USA.

Molecular therapy : the journal of the American Society of Gene Therapy
|December 11, 2025
PubMed
概括

基因纠正的调节性T细胞 (Tregs) 为IL2RA缺乏症,一种罕见的自身免疫性疾病提供了潜在的新疗法. 这种方法使用CRISPR-Cas9来修复基因突变,创建功能Tregs,比目前的选择更安全的治疗方法.

关键词:
克里斯普尔是什么意思?克里斯普尔是什么意思?这就是IL2RA.临床制造 临床制造 临床制造基因纠正 基因纠正免疫的先天错误 免疫的先天错误非病毒性非病毒性.罕见病是一种罕见的疾病.监管性T细胞是什么

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