- VernacularTitle:遗传性血管性水肿基因治疗研究进展
- Author:
Yuqi SHI
1
;
Juan LIU
1
;
Yuxiang ZHI
1
Author Information
- Publication Type:Journal Article
- Keywords: hereditary angioedema; gene therapy; research progress
- From: JOURNAL OF RARE DISEASES 2025;4(3):294-301
- CountryChina
- Language:Chinese
-
Abstract:
Hereditary angioedema (HAE) is a rare autosomal dominant genetic disorder characterized by recurrent and unpredictable episodes of edema. Currently, there is no effective cure available, which seriously affects patients′ quality of life. Gene therapy intervenes in the disease at the genetic level, offering new perspectives for the treatment of HAE. This article summarizes the current research progress in gene therapy for HAE, including gene editing, transgenic therapy, and RNA therapy. It aims to draw the attention of researchers in this field, and provide references and insights for clinicians.

