| 发明名称 |
CHEMICALLY MODIFIED CYSTATHIONINE BETA-SYNTHASE ENZYME FOR TREATMENT OF HOMOCYSTINURIA |
| 摘要 |
The invention provides reagents and methods for enzyme replacement therapy using chemically modified species of human cystathionine β-synthase (CBS) to treat homocystinuria and other related diseases and disorders. |
| 申请公布号 |
US2014212403(A1) |
申请公布日期 |
2014.07.31 |
| 申请号 |
US201313803804 |
申请日期 |
2013.03.14 |
| 申请人 |
THE REGENTS OF THE UNIVERSITY OF COLORADO |
发明人 |
KRAUS Jan P;MAJTAN Thomas;BUBLIL Erez |
| 分类号 |
C12N9/96;A61K45/06;A61K38/51 |
主分类号 |
C12N9/96 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A pharmaceutical composition comprising an isolated carboxyl-terminal truncated cystathionine-β-synthase (CBS) polypeptide comprising the amino acid sequence of SEQ ID NO: 2, wherein said CBS protein is a chemically or enzymatically cleaved or genetically engineered truncate thereof, that is covalently linked to at least one water-soluble polymer or mixture of such molecules. |
| 地址 |
Denver CO US |
