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Neonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I
Neonatal bone marrow transplantation (BMT) could offer a novel therapeutic opportunity for genetic disorders by providing sustainable levels of the missing protein at birth, thus preventing tissue damage. We tested this concept in mucopolysaccharidosis type I (MPS IH; Hurler syndrome), a lysosomal s...
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| 發表在: | Blood |
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| Main Authors: | , , , , , , , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
American Society of Hematology
2015
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4351510/ https://ncbi.nlm.nih.gov/pubmed/25298037 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2014-06-581207 |
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