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Updated: Jun 8, 2025

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在多囊性病中基因治疗的前景

Anubhav Chakraborty1,2, Alan S L Yu1,2

  • 1The Jared Grantham Kidney Institute.

Current opinion in nephrology and hypertension
|November 5, 2024
PubMed
概括

基因疗法在治疗多囊性病 (PKD) 方面表现有前途. 研究人员正在探索基因编辑和其他策略,以减少囊的形成和改善功能,尽管仍然存在挑战.

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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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