REDUCED IMMUNOGENIC PROTEINS FOR LYSOSOMAL STORAGE DISORDERS

    公开(公告)号:US20200032229A1

    公开(公告)日:2020-01-30

    申请号:US16599730

    申请日:2019-10-11

    IPC分类号: C12N9/16

    摘要: Disclosed are methods and compositions for reduced immunogenic proteins used in enzyme replacement therapy for lysosomal storage disorders. More specifically disclosed are genetically engineered variants of N-acetylgalactosamine-6-sulfatase (GALNS), which are less immunogenetic then wild-type GALNS, but maintain enzymatic activity, and may be used to treat Mucopolysaccharidosis IVA (Morquio A disease, MPS IVA).