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Updated: Jul 21, 2026

Efficient Generation of hiPSC Neural Lineage Specific Knockin Reporters Using the CRISPR/Cas9 and Cas9 Double Nickase System
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四个人类NAD(P) HX表皮酶 (NAXE) 淘汰 iPSC 线的生成和表征

Tim Sikora1, Myrto Patraskaki2, Sara Howden3

  • 1Murdoch Children's Research Institute, Parkville, Victoria, Australia; Department of Paediatrics, University of Melbourne, Parkville, Victoria, Australia.

Stem cell research
|August 2, 2025
PubMed
概括

研究人员创建了NAXE缺陷的细胞系,以建模PEBEL1,这是一种罕见的婴儿神经代谢障碍,由NAD(P) HX表皮酶 (NAXE) 的致病变体引起. 这种模型有助于研究疾病机制,并开发治疗这种严重疾病的疗法.

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相关概念视频

In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.