Recombinant human acid beta-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 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.
Reggi
Serena
S
Plantechno srl, Via Staffolo 60, Vicomoscano, 26040 Cremona, Italy.
Marchetti
Stefano
S
Patti
Tamara
T
De Amicis
Francesca
F
Cariati
Roberta
R
Bembi
Bruno
B
Fogher
Corrado
C
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*
D010940:Plant Proteins; D011134:Polysaccharides; D011993:Recombinant Fusion Proteins; D005962:Glucosylceramidase