A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II
Molecular therapy(2018)
Key words
enzyme replacement therapy,blood-brain barrier,iduronate-2-sulfatase,mucopolysaccharidosis II,glycosaminoglycans,transferrin receptor,lysosomal storage disease
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