Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism

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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Bibliographic Details
Main Authors: Emily Y. Tan, Jaap Jan Boelens, Simon A. Jones, Robert F. Wynn
Format: Article
Language:English
Published: Frontiers Media S.A. 2019-10-01
Series:Frontiers in Pediatrics
Subjects:
hematopoietic stem cell transplantation
bone marrow transplant
inborn errors of metabolism
lysosomal storage disease
peroxisomal disease
mitochondrial disease
Online Access:https://www.frontiersin.org/article/10.3389/fped.2019.00433/full

Internet

https://www.frontiersin.org/article/10.3389/fped.2019.00433/full

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