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
Main Authors: Pievani, Alice, Azario, Isabella, Antolini, Laura, Shimada, Tsutomu, Patel, Pravin, Remoli, Cristina, Rambaldi, Benedetta, Valsecchi, Maria Grazia, Riminucci, Mara, Biondi, Andrea, Tomatsu, Shunji, Serafini, Marta
格式: Artigo
語言:Inglês
出版: American Society of Hematology 2015
主題:
Transplantation
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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