Optimization of the production of a human enzyme from transgenic tobacco plants grown in greenhouse.

The recombinant enzyme alpha-mannosidase was recently expressed in stably transformed tobacco plants by Agrobacterium-mediated transformation (De Marchis et al., Plant Biotechnol J. 2011, 9:1061). The aim of this study is to develop an enzyme replacement therapy (ERT) for alpha-mannosidosis which is a rare lysosomal storage disease that leads to mental and physical deterioration. This pathology is due to progressive accumulation of undegraded oligosaccharides inside lysosomes and the cause of the disease is the deficiency of the alpha-mannosidase enzyme, which normally cleaves alpha-linked mannose residues from glycoproteins during their ordered degradation. We are trying both to optimized the production of the human enzyme from transgenic tobacco plants grown in greenhouse and to further evaluate the enzyme functional activity in knock-out alpha-mannosidosis mice.