Generation and characterization of recombinant feline beta-galactosidase for preclinical enzyme replacement therapy studies in GM1 gangliosidosis

Metab Brain Dis. 2008 Jun;23(2):161-73. doi: 10.1007/s11011-008-9086-5. Epub 2008 Apr 18.

Abstract

Lysosomal beta-galactosidase is required for the degradation of GM1 ganglioside and other glycolipids and glycoproteins with a terminal galactose moiety. Deficiency of this enzyme leads to the lysosomal storage disorder, GM1 gangliosidosis, marked by severe neurodegeneration resulting in premature death. As a step towards preclinical studies for enzyme replacement therapy in an animal model of GM1 gangliosidosis, a feline beta-galactosidase cDNA was cloned into a mammalian expression vector and subsequently expressed in Chinese hamster ovary (CHO-K1) cells. The enzyme secreted into culture medium exhibited specific activity on two synthetic substrates as well as on the native beta-galactosidase substrate, GM1 ganglioside. The enzyme was purified from transfected CHO-K1 cell culture medium by chromatography on PATG-agarose. The affinity-purified enzyme preparation consisted mainly of the protein with approximate molecular weight of 94 kDa and displayed immunoreactivity with antibodies raised against a 16-mer synthetic peptide corresponding to C-terminal amino acid sequence deduced from the feline beta-galactosidase cDNA.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antibody Specificity / immunology
  • CHO Cells
  • Cats
  • Chromatography, Agarose
  • Cloning, Molecular / methods
  • Cricetinae
  • Cricetulus
  • Culture Media, Conditioned / chemistry
  • DNA, Complementary / genetics
  • Disease Models, Animal
  • G(M1) Ganglioside / biosynthesis*
  • G(M1) Ganglioside / genetics
  • Gangliosidosis, GM1 / enzymology*
  • Gangliosidosis, GM1 / genetics
  • Gangliosidosis, GM1 / therapy
  • Genetic Therapy / methods*
  • Genetic Vectors / genetics
  • Molecular Weight
  • Protein Structure, Tertiary / physiology
  • Recombinant Proteins / genetics
  • Recombinant Proteins / isolation & purification*
  • Recombinant Proteins / metabolism
  • Transfection / methods
  • beta-Galactosidase / genetics
  • beta-Galactosidase / isolation & purification*
  • beta-Galactosidase / metabolism

Substances

  • Culture Media, Conditioned
  • DNA, Complementary
  • Recombinant Proteins
  • G(M1) Ganglioside
  • beta-Galactosidase