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Updated: Jan 14, 2026

Large Animal Model for Evaluating the Efficacy of the Gene Therapy in Ischemic Heart
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Large Animal Model for Evaluating the Efficacy of the Gene Therapy in Ischemic Heart

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开发两种体外ND1-LHON模型,用于评估基因治疗的有效性.

Xin Li1, Jun Yuan1, Zhang Chen1

  • 1Department of Ophthalmology, Taihe Hospital, Hubei University of Medicine, Shiyan, Hubei Province, People's Republic of China.

Investigative ophthalmology & visual science
|October 23, 2025
PubMed
概括

这项研究开发了与NADH-ubiquinone氧化降解酶链1 (ND1) 相关的Leber遗传性视觉神经病变 (LHON) 的体外模型,并测试了复合腺相关病毒 (AAV) 中介的ND1基因疗法. 基因疗法在LHON模型中成功恢复了线粒体功能,显示了治疗潜力.

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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.
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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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