GlcNAc phosphotransferase of the lysosomal targeting pathway
The present invention provides nucleotide and amino sequences of the lysosomal targeting pathway enzyme GlcNAc-phosphotransferase, methods of producing and methods of purifying this enzyme.
1. A pharmaceutical composition suitable for the treatment of a human patient having Pompe disease comprising recombinant human alpha-glucosidase, wherein said composition has an average of at least 5 mannose-6-phosphate residues per molecule human recombinant alpha-glucosidase and a pharmaceutically acceptable carrier.
2. A pharmaceutical composition suitable for the treatment of a human patient having Pompe disease comprising recombinant human alpha-glucosidase, wherein said composition has an average of about at least 6% bis-phosphorylated oligosaccharides per molecule of human recombinant alpha glucosidase and a pharmaceutically effective carrier.