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Updated: Jun 27, 2025

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活体基因编辑和细胞疗法治疗遗传性铁血病1型

Ilayda Ates1, Callie Stuart1, Tanner Rathbone1

  • 1Department of Bioengineering, Clemson University, Clemson, South Carolina, USA.

Hepatology communications
|April 26, 2024
PubMed
概括

这项研究开发了一种使用CRISPR-Cas9和细胞疗法的ex vivo基因编辑协议,用于治疗小鼠1型遗传性铁血病 (HT1). 优化的方法成功地纠正了小鼠的疾病指标,证明了对HT1.1的潜在治疗方法.

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