发明名称 COMBINATION THERAPY FOR TREATING PROTEIN DEFICIENCY DISORDER
摘要 <P>PROBLEM TO BE SOLVED: To provide a method for increasing the stability of the purified proteins. <P>SOLUTION: There is provided a method for improving protein replacement therapy by combining protein replacement therapy with active site-specific chaperone (ASSC) to increase the stability and efficiency of the protein being administered, wherein the purified protein is an enzyme and the active site-specific chaperone is a reversible competitive inhibitor of the enzyme. The enzyme is an enzyme associated with a lysosomal storage disorder, such as &alpha;-galactosidase A or &beta;-glucocerebrosidase. The reversible competitive inhibitor is a compound selected from the group consisting of 1-deoxygalactonojirimycin, &alpha;-allo-homonojirimycin, &alpha;-galacto-homonojirimycin, &alpha;-1-C-butyl-deoxynojirimycin, calystegine A3, calystegine B2, N-methyl-calystegine A3, and N-methyl-calystegine B2. <P>COPYRIGHT: (C)2012,JPO&INPIT
申请公布号 JP2012107020(A) 申请公布日期 2012.06.07
申请号 JP20120000271 申请日期 2012.01.04
申请人 MOUNT SINAI SCHOOL OF MEDICINE OF NEW YORK UNIV 发明人 FAN JIAN-QIANG
分类号 A61K38/00;A61K;A61K9/08;A61K9/19;A61K38/43;A61K38/46;A61K38/47;A61K47/22;A61K47/26;C12N9/24;C12N9/40;C12N9/99 主分类号 A61K38/00
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