Recombinant human acid β-glucosidase stored in tobacco seed is stable, active and taken up by human fibroblasts

Gaucher disease, the most common genetic lysosomal disorder, is caused by the lack of functional acid β-glucosidase (GCase) and is currently treated at a very high cost by enzyme replacement therapy. In an attempt to provide a safe and cost-effective production system, human placental GCase was produced and purified from transgenic tobacco seeds. Plant-derived recombinant GCase was found to be enzymatically active, uptaken by human fibroblasts and free of immunogenic xylose and fucose residues. This report demonstrates the potential of plant bioreactors in the large-scale production of injectable proteins required for lifelong therapy.