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

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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.

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Authors and Affiliations

  1. Plantechno srl, Via Staffolo 60, Vicomoscano, 26040, Cremona, Italy

    Serena Reggi

  2. Dipartimento di Scienze Agrarie e Ambientali, Università di Udine, Via delle Scienze 208, Udine, 33100, Italy

    Stefano Marchetti & Francesca De Amicis

  3. TransPharma srl, Area Science Park, Padriciano 99, Trieste, 34137, Italy

    Tamara Patti

  4. Laboratorio di Malattie Rare, Istituto di Ricerca e Cura a Carattere Scientifico “Burlo Garofolo”, Via dell’Istria 65/1, Trieste, 34137, Italy

    Roberta Cariati & Bruno Bembi

  5. Istituto di Botanica e Genetica Vegetale, Università Cattolica del S. Cuore, Via Emilia Parmense 84, Piacenza, 29100, Italy

    Corrado Fogher

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  1. Serena Reggi
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  2. Stefano Marchetti
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  7. Corrado Fogher
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Correspondence to Corrado Fogher.

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Reggi, S., Marchetti, S., Patti, T. et al. Recombinant human acid β-glucosidase stored in tobacco seed is stable, active and taken up by human fibroblasts. Plant Mol Biol 57, 101–113 (2005). https://doi.org/10.1007/s11103-004-6832-x

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  • DOI: https://doi.org/10.1007/s11103-004-6832-x

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