GLA Human

Alpha-Galactosidase is a homodimeric glycoprotein, meaning it consists of two identical subunits. Each subunit is responsible for releasing terminal alpha-galactosyl moieties from glycolipids and glycoproteins . The enzyme is lysosomal, meaning it is located within the lysosomes of cells, where it aids in the degradation of glycolipids .

Recombinant human alpha-Galactosidase is produced using Chinese Hamster Ovary (CHO) cell lines. The recombinant form is designed to mimic the natural enzyme’s structure and function, ensuring it can effectively hydrolyze substrates like 4-methylumbelliferyl-alpha-D-galactopyranoside . The recombinant enzyme is often tagged with a C-terminal 6-His tag to facilitate purification and detection .

One of the primary clinical applications of recombinant alpha-Galactosidase is in the treatment of Fabry disease. Fabry disease is a genetic disorder caused by a deficiency in alpha-Galactosidase, leading to the accumulation of globotriaosylceramide in various tissues . Enzyme replacement therapy (ERT) with recombinant alpha-Galactosidase helps to reduce this accumulation, alleviating symptoms and improving the quality of life for patients .

Recombinant alpha-Galactosidase is typically supplied as a filtered solution in Tris and NaCl. It is crucial to store the enzyme at -20 to -70°C to maintain its stability and avoid repeated freeze-thaw cycles . Under sterile conditions, the enzyme remains stable for up to six months from the date of receipt .