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

Recombinant human acid β-glucosidase stored in tobacco seed is stable, active and taken up by... 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. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Plant Molecular Biology Springer Journals

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

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Publisher
Kluwer Academic Publishers
Copyright
Copyright © 2005 by Springer
Subject
Life Sciences; Biochemistry, general; Plant Sciences; Plant Pathology
ISSN
0167-4412
eISSN
1573-5028
D.O.I.
10.1007/s11103-004-6832-x
Publisher site
See Article on Publisher Site

Abstract

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.

Journal

Plant Molecular BiologySpringer Journals

Published: Nov 26, 2004

References

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