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BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2019)
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Elisabeth Jameson et al.
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Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model
Ilaria Visigalli et al.
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Vassili Valayannopoulos et al.
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Mucopolysaccharidosis I: Management and Treatment Guidelines
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Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure
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The ocular features of the mucopoly-saccharidoses
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