- 专利标题: REDUCED IMMUNOGENIC PROTEINS FOR LYSOSOMAL STORAGE DISORDERS
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申请号: US15411797申请日: 2017-01-20
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公开(公告)号: US20180073000A1公开(公告)日: 2018-03-15
- 发明人: Adriana Montaño-Suarez , Angela Sosa-Molano
- 申请人: Saint Louis University, a non-profit organization Saint Louis University
- 主分类号: C12N9/16
- 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).
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