Saint Louis University; a non-profit organization Saint Louis University
发明人:
Adriana Montaño-Suarez,Angela Sosa-Molano
申请号:
US15411797
公开号:
US10472615B2
申请日:
2017.01.20
申请国别(地区):
US
年份:
2019
代理人:
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).