earticle

영어

Gaucher's disease is a metabolic disorder in which a functional deficiency of an enzyme occurs due to a mutation in the gene encoding glucocerebrosidase (GC), resulting in multiple organ malfunctions. Gaucher's disease is being treated with enzyme replacement therapy, which compensates for the enzyme deficiency by administering activated GCs produced in mammalian cells. However, there is a potential of contamination by pathogenic viruses or prions in the mammalian cell-based manufacturing method, as well as a high production cost. In this study, GCs were expressed in plants that produce customized N-glycans to overcome the limitations of the mammalian cell-based production system and to produce a safe treatment drug for Gaucher's disease. A human cDNA library was used to clone a region expressing a GC gene, and sequence analysis was performed. In addition, a binary vector was created and introduced into plants using Agrobacterium to insert the sequence-confirmed GC gene. Plants into which the GC gene was introduced were selected using an antibiotic-containing media, and the presence or absence of the GC gene introduction into the transformed plants was confirmed using polymerase chain reaction (PCR). Furthermore, proteins were collected from the plant leaves into which the gene was inserted, and GC expression levels in the transformed plants were measured using a GC antibody. Finally, a single cloned plant was selected through the antibiotic-resistant isolate ratio. These findings suggest that employing plants, it is possible to overcome the disadvantages of the present mammalian cell-based manufacturing method, decreasing the production cost of pricey Gaucher's disease therapy, and effectively generating safer enzyme treatment.