Expression and purification of a human, soluble Arylsulfatase A for Metachromatic Leukodystrophy enzyme replacement therapy

J Biotechnol. 2005 May 25;117(3):243-51. doi: 10.1016/j.jbiotec.2005.01.018.

Abstract

The production of active Arylsulfatase A is a key step in the development of enzyme replacement therapy for Metachromatic Leukodystrophy. To obtain large amounts of purified Arylsulfatase A for therapeutic use, we combined a retroviral expression system with a versatile and rapid purification protocol that can easily and reliably be adapted to high-throughput applications. The purification method consists of an initial ion-exchange DEAE-cellulose chromatography step followed by immuno-affinity purification using a polyclonal antibody against a 29-mer peptide of the Arylsulfatase A sequence. Immuno-adsorbed protein was eluted with a combination of acidic pH and an optimal concentration of the 29-mer peptide. This protocol reproducibly yielded approximately 100 microg of >99% pure human Arylsulfatase A, corresponding to 152 mU of enzyme activity, per liter of culture medium with properties similar to those of human non-recombinant protein.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Blotting, Western
  • Cell Extracts
  • Cell Line
  • Cells, Cultured
  • Cerebroside-Sulfatase / analysis
  • Cerebroside-Sulfatase / genetics
  • Cerebroside-Sulfatase / isolation & purification*
  • Cerebroside-Sulfatase / metabolism*
  • Cerebroside-Sulfatase / therapeutic use*
  • Electrophoresis, Polyacrylamide Gel
  • Genetic Vectors
  • Humans
  • Isoelectric Focusing
  • Leukodystrophy, Metachromatic / enzymology*
  • Leukodystrophy, Metachromatic / therapy*
  • Mice
  • Mice, Knockout
  • Oligodendroglia / cytology
  • Recombinant Proteins / metabolism
  • Retroviridae / genetics
  • Solubility
  • Substrate Specificity
  • Transduction, Genetic

Substances

  • Cell Extracts
  • Recombinant Proteins
  • Cerebroside-Sulfatase