| 发明名称 |
METHODS AND COMPOSITIONS FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES |
| 摘要 |
Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of a lysosomal storage disease. |
| 申请公布号 |
US2014017212(A1) |
申请公布日期 |
2014.01.16 |
| 申请号 |
US201313839336 |
申请日期 |
2013.03.15 |
| 申请人 |
SANGAMO BIOSCIENCES, INC. |
发明人 |
REBAR EDWARD J. |
| 分类号 |
A61K38/46;A61K48/00 |
主分类号 |
A61K38/46 |
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