申请人:biOasis Technologies, Inc.
公开号:US10392605B2
公开(公告)日:2019-08-27
Provided are fusion proteins between p97 (melanotransferrin) and iduronate-2-sulfatase (IDS), and related compositions and methods of use thereof, for instance, to facilitate delivery of IDS across the blood-brain barrier (BBB) and/or improve its tissue penetration in CNS and/or peripheral tissues, and thereby treat and/or diagnose Hunter Syndrome (Mucopolysaccharidosis type II; MPS II) and related lysosomal storage disorders, including those having a central nervous system (CNS) component.
本研究提供了 p97(黑色素转铁蛋白)和伊糖醛酸-2-硫酸酯酶(IDS)之间的融合蛋白及其相关组合物和使用方法,例如,用于促进 IDS 穿过血脑屏障(BBB)和/或改善其在中枢神经系统和/或外周组织中的组织渗透,从而治疗和/或诊断亨特综合征(粘多糖病 II 型;MPS II)和相关溶酶体贮积症,包括具有中枢神经系统(CNS)成分的溶酶体贮积症。