发明名称 |
Compositions and methods for treating lysosomal storage disease |
摘要 |
The present invention provides a biologically active human lysosomal enzyme to cells deficient therein comprising introducing into the cells a vector comprising and expressing a transgene encoding the biologically active human lysosomal enzyme, wherein the vector is taken up by the cells, the transgene is expressed therein and biologically active enzyme is produced. |
申请公布号 |
AU1284799(A) |
申请公布日期 |
2000.03.06 |
申请号 |
AU19990012847 |
申请日期 |
1998.10.29 |
申请人 |
GENZYME CORPORATION |
发明人 |
NELSON S YEW;ROBIN J ZIEGLER;SENG H CHENG |
分类号 |
A61K31/00;A61K35/76;A61K38/43;A61K38/47;A61K48/00;A61P3/00;A61P43/00;C12N9/40 |
主分类号 |
A61K31/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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