Abstract
Mucopolysaccharidosis type Ⅱ (MPSⅡ) is a kind of monogenic disease which belongs to X-linked recessive inheritance. All MPSⅡ patients have a deficiency of iduronate-2-sulfatase (IDS), which results in the accumulation of glycosaminoglycans (GAG) in lysosomes. As more and more GAG accumulates, patients will develop a series of dysfunctions. Till now, MPSⅡ therapies include enzyme replacement therapy, hematopoietic stem cell transplantation and gene therapy. With the development of medical treatment, MPSⅡ patients will eventually be cured some day. Key words: Mucopolysaccharidosis type Ⅱ; X-linked recessive inheritance; Iduronate-2-sulfatase; Glycosaminoglycan; Gene therapy
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