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Natural sciences
- Systems biology not elsewhere classified
We propose in this project to produce variants (glycoforms) of therapeutic proteins linked to very specific sugar structures (N-glycans).
Through the structure of these glycans, it is possible to target the therapeutic proteins to the exact location in the body where they are
needed to exert their therapeutic function. The project builds on the previous and ongoing development in our laboratory of yeast-based
production technology for such specific glycoforms. Within this larger research programme, we are now in a position to test these
glycoforms in experimental disease models and have assembled a team of scientists to this effect. With this grant application, we request
an operational budget to enable these rather expensive experiments. We focus on two types of diseases. First, we aim at strongly
improving the therapeutic efficacy of Enzyme Replacement Therapy for inherited lysosomal storage diseases, while substantially
reducing the enormous current cost of such therapy. Second, we aim at delivering the liver-protective cytokine IL-22 specifically to the
hepatocyte cell surface, as an experimental therapy for progressive non-viral chronic liver disease, in particular Non-Alcoholic
SteatoHepatitis (NASH), the prevalence of which is rapidly increasing worldwide.