Last Updated:2025/11/28

Researchers are developing enzyme replacement therapies to treat Hurler syndrome, a genetic disorder that causes the buildup of glycosaminoglycans due to a deficiency of alpha-L iduronidase.

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Researchers are developing enzyme replacement therapies to treat Hurler syndrome, a genetic disorder that causes the buildup of glycosaminoglycans due to a deficiency of alpha-L iduronidase.

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研究者たちは、α-L-イドゥロナーゼ欠損によりグリコサミノグリカンが蓄積する遺伝性疾患であるムコ多糖症I型を治療するための酵素補充療法を開発している。

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