Some of the worst human genetic diseases are caused by missing enzymes. They can, however, be easily substituted – but only if the substitute is human-like and efficient. Moss plants are perfectly suited to successful humanization of enzymes. Capitalizing on this, we develop enzymes that are tolerated by the human immune system and easily taken up into the relevant cells, making a world of a difference to patients.
For an injected replacement enzyme in the blood stream to be taken up into the cells, its N-glycan structure (sugar residues on the surface) is crucial. The right pattern and homogeneity determine the therapeutic success. Studies have shown that 95 % of moss N-glycans are relevant for best uptake of the recombinant enzyme into the cell and for optimal organ distribution.