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A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II

Molecular therapy(2018)

Cited 132|Views70
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Key words
enzyme replacement therapy,blood-brain barrier,iduronate-2-sulfatase,mucopolysaccharidosis II,glycosaminoglycans,transferrin receptor,lysosomal storage disease
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