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METHOD OF TREATING MUCOPOLYSACCHARIDOSIS TYPE IVA

机译:治疗粘性多种型IVA的方法

摘要

Disclosed are methods and compositions for determining immunodominant peptides of target enzymes used in enzyme replacement therapy for lysosomal storage disorders. More specifically disclosed are immunodominant peptides for N-acetylgalactosamine-6-sulfatase (GALNS). Also disclosed are methods of inducing oral tolerance towards a target enzyme through oral administration of immunodominant peptides prior to commencing enzyme replacement therapy. More specifically disclosed is a method of inducing oral tolerance for GALNS, by orally administering specific immunodominant peptides for GALNS; in subjects suffering from mucopolysaccharidosis type IVA prior to commencing enzyme replacement therapy using GALNS.
机译:公开了用于确定用于酶储存障碍的酶替代疗法中使用的靶酶的免疫蛋白肽的方法和组合物。 更具体地公开的是N-乙酰甘油蛋胺-6-硫酸酯酶(GALNS)的免疫肿瘤。 还公开了通过在开始酶替代疗法之前口服免疫蛋白肽来诱导靶向靶酶的口服耐受的方法。 更具体地公开的是通过口服给GALNS口服施用特异性免疫染色肽对GALN进行口服耐受的方法; 在使用Galns开始酶替代疗法之前患有IVA患有IVA的受试者。

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