公开(公告)号：US20050064539A1

公开(公告)日：2005-03-24

申请号：US10480790

申请日：2002-06-14

申请人： Yasunori Chiba ,  Yoshifumi Jigami ,  Hitoshi Sakuraba ,  Kazuo Kobayashi ,  Makoto Takeuchi ,  Yoriko Takeuchi

发明人： Yasunori Chiba ,  Yoshifumi Jigami ,  Hitoshi Sakuraba ,  Kazuo Kobayashi ,  Makoto Takeuchi ,  Yoriko Takeuchi

IPC分类号： C12N15/09 ,  A61K36/064 ,  A61K38/00 ,  A61K38/17 ,  A61P9/00 ,  A61P9/14 ,  A61P13/12 ,  A61P17/00 ,  A61P19/00 ,  A61P27/02 ,  A61P43/00 ,  C08B37/00 ,  C12N9/10 ,  C12N9/40 ,  C12P21/00 ,  C12P21/06 ,  C12N1/18

CPC分类号： C12P21/005 ,  A61K36/064 ,  A61K38/17 ,  C12N9/1051 ,  A61K2300/00

摘要： A process for producing a lysosomal enzyme having a mannose-6-phosphate-containing acidic sugar chain, wherein the process comprising: culturing in a medium yeast cells obtained by introducing a lysosomal enzyme gene into a sugar chain biosynthetic enzyme gene mutant strain of yeast, collecting a lysosomal enzyme having a phosphate-containing sugar chain from the culture, and then treating the enzyme with α-mannosidase; and pharmaceutical compositions for treatment of human lysosomal enzyme deficiencies produced by the process. The genetic engineering technique using the yeast according to the present invention allows large-amount and high-purity production of a glycoprotein having a phosphate-containing acidic sugar chain which can serve as a labeling marker for transporting into lysosomes in cells of mammals such as human. The glycoprotein having a phosphate-containing acidic sugar chain according to the invention may be utilized as a drug effective in treatment of human lysosomal enzyme deficiencies, etc.

摘要翻译： 一种生产含有甘露糖-6-磷酸的酸性糖链的溶酶体酶的方法，其中所述方法包括：在通过将溶酶体酶基因导入酵母的糖链生物合成酶基因突变菌株获得的培养基中培养酵母， 从培养物中收集含有含磷酸盐糖链的溶酶体酶，然后用α-甘露糖苷酶处理酶; 以及用于治疗由该方法产生的人溶酶体酶缺陷的药物组合物。 使用根据本发明的酵母的基因工程技术允许大量和高纯度产生具有含磷酸盐的酸性糖链的糖蛋白，其可以用作在诸如人类的哺乳动物的细胞中输送到溶酶体的标记标记 。 具有根据本发明的含磷酸盐酸性糖链的糖蛋白可以用作治疗人溶酶体酶缺陷等的药物。