发明名称 |
COMPOSITIONS AND METHODS FOR TREATING HYPOPHOSPHATASIA |
摘要 |
The present invention provides compositions and methods for use in enzyme replacement therapy. The inventors disclose a method of producing membrane bound enzymes in an active soluble form by eliminating the glycosylphosphatidylinositol (GPI) membrane anchor. In particular the inventors disclose a soluble active form of the membrane bound enzyme TNSALP which they produced by deleting the GPI anchor single peptide sequence. They have further shown that this composition is useful for treatment of hypophosphatasia. The inventors also disclose oligo acid amino acid variants thereof which specifically target bone tissue.
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申请公布号 |
US2011250187(A1) |
申请公布日期 |
2011.10.13 |
申请号 |
US201113071445 |
申请日期 |
2011.03.24 |
申请人 |
SAINT LOUIS UNIVERSITY |
发明人 |
TOMATSU SHUNJI;SLY WILLIAM;GRUBB JEFFREY;NISHIOKA TATSUO;MIYAMOTO KEN-ICHI;YAMAGUCHI SEIJI |
分类号 |
A61K35/54;A61K38/46;A61K48/00;A61P3/00;C12N9/16 |
主分类号 |
A61K35/54 |
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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