Inventors:
Shunji Tomatsu - Saint Louis MO, US
William S Sly - Saint Louis MO, US
Jeffrey H Grubb - Saint Louis MO, US
Tatsuo Nishioka - Kanazawa, JP
Ken-ichi Miyamoto - Kanazawa, JP
Seiji Yamaguchi - Izumo, JP
Assignee:
Saint Louis University - Saint Louis MO
International Classification:
A61K 38/43, A61P 19/08, C12N 9/00, C12P 21/02
US Classification:
424 941, 514 12, 530350, 435 691, 435183
Abstract:
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.