遗传性血管性水肿基因治疗研究进展

Research Progress in Gene Therapy for Hereditary Angioedema

  • 摘要: 遗传性血管性水肿(hereditary angioedema,HAE)是一种罕见的、以反复发作、不可预测的水肿为主要表现的常染色体显性遗传病,目前尚无有效的根治手段,严重影响患者的生活质量。基因治疗从基因层面干预疾病,为HAE的治疗带来了新思路。本文概述了当前基因治疗在HAE领域的研究进展,包含基因编辑、转基因治疗、RNA治疗等内容,旨在引起该领域研究者的关注,为临床医生提供参考和借鉴。

     

    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.

     

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