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You are here: Home Research Group Gieselmann Group Matzner

Group Matzner

Dr. Ulrich Matzner

We are interested in therapy approaches for MLD. The N-glycans of ASA harbour mannose 6-phosphate (M6P) residues which bind to M6P-receptors of the plasma membrane mediating endocytosis and lysosomal delivery of the ligand. M6P-dependent endocytosis therefore opens the possibility to treat MLD by substitution therapy.

Using ASA knockout mice as an animal model of MLD, we are testing and optimizing different routes of enzyme delivery (in vivo gene therapy, ex vivo gene therapy, cell therapy, enzyme replacement therapy). For enzyme replacement therapy (ERT) recombinantly expressed ASA is supplied by repeated intravenous injection. Treatment reduces sulfatide storage throughout the nervous system and has a favourable benefit-to-risk ratio leading to the approval of a clinical phase I/II trial.

Attempts to optimize ERT focus on the blood-brain barrier which hampers the transfer of therapeutic enzyme from the circulation to the brain parenchyma. To overcome the BBB more efficiently we are exploiting (read more)...


Frank Matthes          Annika Boeckenhoff
Debora Gerlach          Tilman Schuster
Simeon Knieling  
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