Advances in the treatment of Wilson disease
10.3760/cma.j.issn.1673-4408.2024.03.003
- VernacularTitle:威尔逊病治疗的研究进展
- Author:
Mengqi ZHANG
1
;
Lingfen XU
Author Information
1. 中国医科大学附属盛京医院小儿消化内科，沈阳 110004
- Keywords:
Wilson disease;
Chelator of copper;
Zinc;
Gene therapy
- From:
International Journal of Pediatrics
2024;51(3):153-158
- CountryChina
- Language:Chinese
-
Abstract:
Wilson disease（WD）is an inherited disorder caused by mutations in the ATP7B gene.It is characterized by pathological accumulation of copper in the organs throughout the body，especially the liver and brain. This article reviews the latest progress of existing and emerging therapies. At present，the most commonly used treatment methods are oral chelators of copper and zinc agents. Liver transplantation can be used as a treatment for end-stage patients. However，these treatment methods have some shortcomings：many side effects of drugs，poor treatment effect，lifelong treatment，poor patient compliance and so on. At present，the choice of drugs is still in the exploratory stage，and there are no guidelines to specify which patients should receive which treatment. In addition，there are many emerging therapies in different experimental stages，such as new chelators of copper，targeted molecular therapy，gene therapy and cell transplantation. The old methods have many disadvantages and lack large controlled clinical trials，and emerging research is still in its infancy，which are urgent questions.
