The researchers are using tobacco plants to produce human lysosomal enzymes. These enzymes are deficient in individuals with rare lysosomal storage diseases, such as Gaucher's disease. Replacement therapy with recombinant enzymes holds tremendous promise for correcting the severe abnormalities associated with these diseases, says Cramer.
Patients usually receive the treatment every two weeks for life.
CropTech researchers have put the gene for glucocerebrosidase into tobacco and shown that an enzymatically active enzyme is produced in tobacco leaves. In the plants making glucocerebrosidase at the highest levels, leaves from a single tobacco plant could make enough enzyme for one dose. Many human proteins, like glucocerebrosidase, also contain sugars called glycans as well as amino acids. Tobacco puts glycans on the right spot on the human protein but the exact composition of the sugars may be different, making the enzyme potentially inappropriate for use in humans.