| 发明名称 |
METHODS AND COMPOSITIONS FOR REGULATION OF METABOLIC DISORDERS |
| 摘要 |
Cells and methods of using these cells for expressing a transgene expressing a protein that is aberrantly expressed in a metabolic disorders from a safe harbor locus. Gene therapy holds enormous potential for a new era of human therapeutics. These methodologies will allow treatment for conditions that have not been addressable by standard medical practice. |
| 申请公布号 |
CA2892448(A1) |
申请公布日期 |
2014.06.12 |
| 申请号 |
CA20132892448 |
申请日期 |
2013.12.04 |
| 申请人 |
SANGAMO BIOSCIENCES, INC.;THE CHILDREN'S HOSPITAL OF PHILADELPHIA |
发明人 |
GREGORY, PHILIP D.;HIGH, KATHERINE A. |
| 分类号 |
C12N5/10;A61K48/00;C12N15/09;C12N15/85;C12N15/90 |
主分类号 |
C12N5/10 |
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