Collection of a mobilized peripheral blo
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A. Alex Hofling; Mark S. Sands; Douglas M. Lublin; Gerhard Bauer; Steven Devine
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Article
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2004
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John Wiley and Sons
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English
⚖ 67 KB
## Abstract The effectiveness of bone marrow transplantation for lysosomal storage diseases like mucopolysaccharidosis type VII (MPSVII) suggests that a gene therapy strategy targeting autologous hematopoietic progenitor cells could be successful. Given the severe systemic manifestations of MPSVII