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Updated: Dec 25, 2025

Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins and mRNA into Freshly Isolated Primary Mouse Hepatocytes
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一个冲击,而不是一个疗法:电穿孔揭示了肝细胞基因编辑疗法的疾病特异性限制

Callie Clark1, Menam Pokhrel1, Benjamin Arthur1

  • 1Department of Bioengineering, Clemson University, Clemson, SC 29634, USA.

Biology
|September 4, 2025
PubMed
概括

在家族性高胆固醇血症中,CRISPR- Cas9基因编辑针对P450氧化还原酶 (Cypor) 通过电穿孔和乙氨基选择是不可行的. 甲缺乏会影响胆固醇代谢,从而阻止小鼠的治疗效果.

关键词:
在CRISPR-Cas9中动脉样硬化电穿孔家庭高胆固醇血症基因编辑肝细胞移植肝脏研究

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