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Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism

Hematopoietic stem cell transplantation (HSCT) has been established as an effective therapy for selected inborn errors of metabolism. The success of HSCT in metabolic disease is best exemplified through the treatment of Hurler's syndrome, a lysosomal storage disease. Through the collaborative e...

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Podrobná bibliografie
Vydáno v:Front Pediatr
Hlavní autoři: Tan, Emily Y., Boelens, Jaap Jan, Jones, Simon A., Wynn, Robert F.
Médium: Artigo
Jazyk:Inglês
Vydáno: Frontiers Media S.A. 2019
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6824291/
https://ncbi.nlm.nih.gov/pubmed/31709204
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fped.2019.00433
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