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Induced Pluripotent Stem Cells to Understand Mucopolysaccharidosis. I: Demonstration of a Migration Defect in Neural Precursors

Background: Mucopolysaccharidosis type I-Hurler (MPS1-H) is a severe genetic lysosomal storage disorder due to loss-of-function mutations in the IDUA gene. The subsequent complete deficiency of alpha l-iduronidase enzyme is directly responsible of a progressive accumulation of glycosaminoglycans (GA...

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Detalles Bibliográficos
Autores principales: Lito, Silvin, Sidibe, Adama, Ilmjarv, Sten, Burda, Patricie, Baumgartner, Matthias, Wehrle-Haller, Bernhard, Krause, Karl-Heinz, Marteyn, Antoine
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7761689/
https://www.ncbi.nlm.nih.gov/pubmed/33287330
http://dx.doi.org/10.3390/cells9122593