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Updated: Jun 3, 2025

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对于Bietti晶体 Dystrophy的基因治疗后的安全性和视力结果:非随机临床试验

Xiuju Chen1,2, Xiao Liu3,4,5, Shihe Cui6

  • 1Xiamen Eye Center of Xiamen University, Xiamen, Fujian, China.

JAMA ophthalmology
|January 9, 2025
PubMed
概括

基因疗法显示出对Bietti结晶性缩症 (BCD) 的潜力. 使用编码CYP4V2的腺相关病毒 (AAV) 的第一阶段试验证明了这种严重遗传视网膜病变的患者的安全性和一定程度的视力改善.

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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...
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