黏多糖贮积症Ⅱ型的基因诊疗

doi:10.12372/jcp.2024.23e0682

Abstract

Abstract:

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.

Key words: mucopolysaccharidosis type Ⅱ, gene therapy, clinical trial, adeno-associated virus

CHEN Guoqing, ZHANG Huiwen. Gene therapy for mucopolysaccharidosis type Ⅱ[J].Journal of Clinical Pediatrics, 2024, 42(3): 270-276.

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Gene therapy for mucopolysaccharidosis type Ⅱ

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