METHODS AND COMPOSITIONS FOR INCREASING ENZYME ACTIVITY IN THE CNS
    1.
    发明申请
    METHODS AND COMPOSITIONS FOR INCREASING ENZYME ACTIVITY IN THE CNS 审中-公开
    在CNS中增加酶活性的方法和组合物

    公开(公告)号:WO2015012944A1

    公开(公告)日:2015-01-29

    申请号:PCT/US2014/038660

    申请日:2014-05-19

    Abstract: Provided herein are methods and compositions for treating a subject suffering from an enzyme deficiency in the central nervous system (CNS). The bifunctional fusion antibodies provided herein comprise an antibody to an endogenous blood brain barrier (BBB) receptor and an enzyme deficient in mucopolysaccharidosis III (MPS-III). The fusion antibodies provided herein comprise N- sulfoglucosamine sulfohydrolase (SGSH), alpha-N-acetylgulcosaminidase (NAGLU), heparin-alpha- glucosaminide N-acetyltransferase (HGSNAT), or N-acetylglucosamine-6-sulfatase (GNS). The methods of treating an enzyme deficiency in the CNS comprise systemic administration of a fusion antibody provided herein.

    Abstract translation: 本文提供了用于治疗患有中枢神经系统(CNS)中的酶缺乏的受试者的方法和组合物。 本文提供的双功能融合抗体包含针对内源性血脑屏障(BBB)受体的抗体和缺陷型粘多糖贮积症III(MPS-III)的酶。 本文提供的融合抗体包含N-磺基葡糖胺磺基水解酶(SGSH),α-N​​-乙酰氨基糖苷酶(NAGLU),肝素-α-葡糖胺-N-乙酰转移酶(HGSNAT)或N-乙酰葡糖胺-6-硫酸酯酶(GNS)。 在CNS中治疗酶缺乏症的方法包括全身给予本文提供的融合抗体。

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