黏多糖贮积症Ⅱ型的基因诊疗
Gene therapy for mucopolysaccharidosis type Ⅱ
Received date: 2023-07-25
Online published: 2024-03-06
陈国庆 , 张惠文 . 黏多糖贮积症Ⅱ型的基因诊疗[J]. 临床儿科杂志, 2024 , 42(3) : 270 -276 . DOI: 10.12372/jcp.2024.23e0682
The two major treatments for mucopolysaccharidosis type Ⅱ (MPS Ⅱ) are hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT). The restoration of central nervous system function is limited by these two therapies, although they are successful in controlling the worsening of physical symptoms. The main limitation is that the blood-brain barrier (BBB) prevents large molecules, including enzymes, from entering the brain, which can affect the effectiveness of treatment. Gene therapy is an emerging therapy for MPS Ⅱ. This article will describe the current preclinical research status and ongoing clinical trials of MPS Ⅱ gene therapy to demonstrate the latest advances in this field.
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