Haploidentical stem cell transplantation in two children with mucopolysaccharidosis VI: clinical and biochemical outcome

BACKGROUND: Mucopolysaccharidosis VI (MPS VI) is an autosomal recessive progressive multiorgan disorder due to mutation in the gene encoding the enzyme Arylsulfatase B (ARSB). Dysfunctional ARSB causes lysosomal accumulation of glycosaminoglycans (GAG). Currently, enzyme replacement therapy (ERT) is...

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Detalles Bibliográficos
Autores principales: Jester, Sandra, Larsson, Julia, Eklund, Erik A, Papadopoulou, Domniki, Månsson, Jan-Eric, Békássy, Albert N, Turkiewicz, Dominik, Toporski, Jacek, Øra, Ingrid
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2013
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3766644/
https://www.ncbi.nlm.nih.gov/pubmed/24107440
http://dx.doi.org/10.1186/1750-1172-8-134

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3766644/
https://www.ncbi.nlm.nih.gov/pubmed/24107440
http://dx.doi.org/10.1186/1750-1172-8-134

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