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Liver‐Directed Human Amniotic Epithelial Cell Transplantation Improves Systemic Disease Phenotype in Hurler Syndrome Mouse Model

Mucopolysaccharidosis type 1 (MPS1) is an inherited lysosomal storage disorder caused by a deficiency in the glycosaminoglycan (GAG)‐degrading enzyme α‐l‐iduronidase (IDUA). In affected patients, the systemic accumulation of GAGs results in skeletal dysplasia, neurological degeneration, multiple org...

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Detalles Bibliográficos
Autores principales:	Rodriguez, Natalie S., Yanuaria, Lisa, Parducho, Kevin Murphy R., Garcia, Irving M., Varghese, Bino A., Grubbs, Brendan H., Miki, Toshio
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2017
Materias:	Translational Research Articles and Reviews
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5689764/ https://www.ncbi.nlm.nih.gov/pubmed/28585336 http://dx.doi.org/10.1002/sctm.16-0449

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