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Cell Press, Molecular Therapy - Methods and Clinical Development, (29), p. 439-449, 2023

DOI: 10.1016/j.omtm.2023.05.010

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Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice

This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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