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Published in 2020 at "Science Translational Medicine"
DOI: 10.1126/scitranslmed.aay8980
Abstract: In utero therapy with recombinant enzyme or hematopoietic stem cells in MPS7 mice induces tolerance to missing enzyme and corrects brain microglia. When treating at birth is too late Mucopolysaccharidosis type VII (MPS7) is a…
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Keywords:
mucopolysaccharidosis type;
mps7;
type vii;
mice ... See more keywords