| 发明名称 | Methods and materials for treatment of pompe's disease | ||
| 摘要 | This document relates to molecular complexes having acid alpha glucosidase activity and at least one modification that results in enhanced ability of the molecular complex to be transported to the interior of a mammalian cell. | ||
| 申请公布号 | US9249399(B2) | 申请公布日期 | 2016.02.02 |
| 申请号 | US201313831368 | 申请日期 | 2013.03.14 |
| 申请人 | Oxyrane UK Limited | 发明人 | Vervecken Wouter;Telesphore Alida Maria Piens Kathleen Camilla;Ludo Stout Jan Robert;Pynaert Gwenda Noëlla |
| 分类号 | C12N9/26;C12P21/02;C12P21/06;C07K19/00;C07K14/65;C12N9/62;A61K38/43;A61K38/47;C12N9/44;A61K38/46;A61K38/00 | 主分类号 | C12N9/26 |
| 代理机构 | Fish & Richardson P.C. | 代理人 | Fish & Richardson P.C. |
| 主权项 | 1. A method for making a molecular complex having acid alpha glucosidase (GAA) activity, said method comprising contacting a GAA protein having at least 90% sequence identity to the amino acid sequence set forth in SEQ ID NO:1 with a protease having at least 85% sequence identity to the amino acid sequence set forth in SEQ ID NO:8, wherein said protease cleaves said protein at one or more sites between amino acid 60 and amino acid 65 of SEQ ID NO:1 to produce a molecular complex having GAA activity and comprising at least two polypeptides. | ||
| 地址 | Manchester GB | ||