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ABUNDANT, STABLE PHARMACEUTICAL PROTEINS TO TREAT HUMAN LYSOSOMAL DISEASES, PRODUCED IN SEEDS
Lysosomal diseases represent a wide class of human genetic diseases determined by malfunctions of specific lysosomal enzymes. Lysosomes are the main degradative organelles of animal cells and are of critical importance in degrading macromolecules and in recycling their monomeric components.
Gaucher disease is one of the most common genetic lysosomal disorders, and is caused by a deficiency of glucocerebrosidase. It is currently treatable, but at a very high cost using enzyme replacement therapy. Anderson-Fabry disease, a genetic disease caused by a malfunctioning α-galactosidase, can cause kidney failure in young males.
Plantechno has developed a plant-based production system whereby the human lysosomal enzymes glucoerebrosidase and α-galactosidase A are expressed at high levels specifically in seeds of transformed plants. Seed expression provides a convenient means of stable enzyme storage, greatly extending the storage life and protein yield over leaf-expressed proteins. Protein production is between 0.8-1.0% of total seed protein. Plant-derived recombinant GCase was found to be enzymatically active, uptaken by human fibroblasts and free of immunogenic xylose and fucose residues. Plantechno has a patent on patent covering the production of several human lysosomal enzymes for enzyme therapy available for transformation into target plants, as well as purification methods, verification that recombinant proteins are enzymatically active, taken up by human fibroblasts, and free of immunogenic residues.
Patent and References:
US 2006/0031965 A1. In-seed expression of lysosomal enzymes.
WO 03/073839 A2. In-seed expression of lysosomal enzymes.
"Recombinant human acid ß-glucosidase stored in tobacco seed is stable, active and taken up by human fibroblasts". Plant Molecular Biology (2005) 57:101-113.
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P.Iva. 01080070194