| 摘要 |
PURPOSE: A yeast Pex5p protein functioning as a peroxisome targeting signal 1-specific molecular chaperone is provided to treat metabolic diseases such as Zellweger syndrome and neonatal adrenoleukodystrophy. CONSTITUTION: A yeast Pex5p protein has three WXXXF/Y motifs(W is trpytophane, F is phenylalanine, Y is tyrosine, and X is a random amino acid) and seven TPR domains. The molecular chaperone is activated through the three TPR domains. A pharmaceutical composition for treating metabolic diseases contains the yeast Pex5p proteins. The metabolic diseases are Zellweger syndrome, neonatal adrenaleukodystropy(NALD), and infantile refsum disease(IRD). [Reference numerals] (AA) GST : : yeastPex5p(1-612); (BB) GST : : yeastPex5p(120-612); (CC) GST : : yeastPex5p(204-612); (DD) GST : : yeastPex5p(267-612); (EE) GST : : yeastPex5p(267-524); (FF) GST : : yeastPex5p(267-490); (GG) GST : : yeastPex5p(267-456); (HH) GST : : yeastPex5p(267-412); (II) GST : : yeastPex5p(267-380); (JJ) GST : : yeastPex5p(267-346); (KK) GST : : yeastPex5p(267-311) |
