method for preparing a target conjugate peptide for a recombinant lysosomal enzyme, method for preparing a molecule for enzyme replacement therapy, conjugate, method for treating an individual suffering from a lysosomal storage disease, method for treating an individual suffering from a lysosomal storage, method for treating a patient suffering from pompe disease, fabry disease, and gaucher disease, mps i, mps ii, tay sachs, sandhoff, alpha-mannosidosis, and wohlman, dna sequence, and sequence of amino acids

摘要

abstract “method for preparing a conjugated target peptide for a recombinant lysosomal enzyme, method for preparing a molecule for enzyme replacement therapy, conjugate, method for treating an individual suffering from a lysosomal storage disease, method for treating an individual suffering from a lysosomal storage disease, method for treating a patient suffering from pompe disease, fabry disease, and gaucher disease, mps i, mps ii, mps vii, tay sachs, sandhoff,? -mannosidosis, and wohlman, dna and Methods for Preparing Target Conjugated Peptides for Recombinant Lysosomal Enzymes by Modifying the (n) -terminal amino and one or more lysine residues in recombinant human lysosomal enzymes using a first crosslinking agent to obtain recombinant human lysosomal enzymes are described herein. modified by the first crosslinking agent by modifying the first amino acid within a amino (n) -terminal short binder on an variant igf-2 peptide using a second crosslinker to obtain a variant igf-2 peptide modified by the second crosslinker, and then conjugate the recombinant human lysosomal enzyme modified by the first crosslinker to the peptide igf-2 variant modified by the second crosslinker containing a short binder. Also described herein are synthesized conjugates characterized as having higher affinity for the igf2 / ci-mpr receptor and cell absorption using the methods described herein. Further described are methods for treatment using the described conjugates. 1