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在小鼠基因治疗后,成熟的人类frataxin的表达和处理

Teerapat Rojsajjakul1, Nithya Selvan2, Bishnu De3

  • 1University of Pennsylvania Philadelphia.

Research square
|January 18, 2024
PubMed
概括

使用AAVrh.10hFXN的弗里德里希缺氧基因疗法成功诱导了小鼠心脏和肝脏中的治疗性frataxin (FXN) 蛋白水平. 这种方法显示出治疗弗里德里希性无氧症中的FXN缺乏和线粒体功能障碍的潜力.

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Gene Therapy00:59

Gene Therapy

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 inserted. The...