title: Recombinant human acid beta-glucosidase stored in tobacco seed is stable, active and taken up by human fibroblasts.
abstract: Gaucher disease, the most common genetic lysosomal disorder, is caused by the lack of functional acid beta-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.
authors.affiliation: Plantechno srl, Via Staffolo 60, Vicomoscano, 26040 Cremona, Italy.
authors.forename: Serena
authors.initials: S
authors.lastname: Reggi
authors.forename: Stefano
authors.initials: S
authors.lastname: Marchetti
authors.forename: Tamara
authors.initials: T
authors.lastname: Patti
authors.forename: Francesca
authors.initials: F
authors.lastname: De Amicis
authors.forename: Roberta
authors.initials: R
authors.lastname: Cariati
authors.forename: Bruno
authors.initials: B
authors.lastname: Bembi
authors.forename: Corrado
authors.initials: C
authors.lastname: Fogher
mesh_terms: D015153:Blotting, Western; D005260:Female; D005347:Fibroblasts / Q000378:metabolism*; D005776:Gaucher Disease / Q000201:enzymology / Q000235:genetics / Q000378:metabolism; D005962:Glucosylceramidase / Q000235:genetics / Q000276:immunology / Q000378:metabolism*; D006801:Humans; D016253:Microscopy, Immunoelectron; D020125:Mutation, Missense; D010920:Placenta / Q000378:metabolism; D010940:Plant Proteins / Q000235:genetics / Q000378:metabolism; D030821:Plants, Genetically Modified; D010957:Plasmids / Q000235:genetics; D011134:Polysaccharides / Q000172:deficiency; D011993:Recombinant Fusion Proteins / Q000235:genetics / Q000378:metabolism; D012639:Seeds / Q000235:genetics* / Q000254:growth & development / Q000648:ultrastructure; D014026:Nicotiana / Q000235:genetics*
chemical_list: D010940:Plant Proteins; D011134:Polysaccharides; D011993:Recombinant Fusion Proteins; D005962:Glucosylceramidase