The project named ”High efficiency production of Recombinant Human β-glucocerebrosidase enzyme for Enzyme Replacement Therapy of Gaucher Disease in Pichia pastoris protein expression system”, led by Gebze Technical University Bioengineering Department faculty member Assoc. Prof. Barış Binay and researched by Gebze Technical University Biotechnology Institute faculty member Assoc. Prof. Müge İşleten Hoşoğlu, Tekirdağ Namık Kemal University, Faculty of Medicine, Department of Medical Biology, Instructor Member Dr. Özlem Yalçın Capan, and Aydın Adnan Menderes University Faculty of Medicine Department of Basic Medical Sciences, Prof. Dr. Gizem Donmez Yalcin, was entitled to be supported within the scope of the B-Group R&D project of the Presidency of Turkish Health Institutes (TÜSEB).
Within the scope of the project, it is aimed to produce the enzyme β-glucocerebrosidase, which is used in the treatment of Gaucher disease, which is one of the important rare diseases that has attracted the attention of governments in recent years. Gaucher disease is the most common lysosomal storage disease in humans, due to deficiency of the β-glucocerebrosidase enzyme, resulting in harmful accumulation of fatty glucocerebroside in the spleen, liver, lungs, bone marrow, and brain. Effective enzyme replacement therapy is available for these patients, but production of β-glucocerebrosidase is expensive. The resulting cost makes many patients unable to afford treatment, and healthcare carriers are reluctant to undertake lifelong treatment. This proposed project focuses on the recombinant production of β-glucocerebrosidase enzyme in high yield (high quantity, low cost) using yeast protein expression system.