公开(公告)号：US20180073000A1

公开(公告)日：2018-03-15

申请号：US15411797

申请日：2017-01-20

申请人： Saint Louis University, a non-profit organization Saint Louis University

发明人： Adriana Montaño-Suarez ,  Angela Sosa-Molano

IPC分类号： C12N9/16

CPC分类号： C12N9/16 ,  A61K38/00 ,  C12Y301/06004

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).

公开(公告)号：US10472615B2

公开(公告)日：2019-11-12

申请号：US15411797

申请日：2017-01-20

申请人： Saint Louis University, a non-profit organization Saint Louis University

发明人： Adriana Montaño-Suarez ,  Angela Sosa-Molano

IPC分类号： C12N9/16 ,  A61K38/00

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).