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Impaired neural differentiation of MPS IIIA patient induced pluripotent stem cell-derived neural progenitor cells

Mucopolysaccharidosis type IIIA (MPS IIIA) is characterised by a progressive neurological decline leading to early death. It is caused by bi-allelic loss-of-function mutations in SGSH encoding sulphamidase, a lysosomal enzyme required for heparan sulphate glycosaminoglycan (HS GAG) degradation, that...

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Detalles Bibliográficos
Autores principales: Lehmann, Rebecca J., Jolly, Lachlan A., Johnson, Brett V., Lord, Megan S., Kim, Ha Na, Saville, Jennifer T., Fuller, Maria, Byers, Sharon, Derrick-Roberts, Ainslie L.K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8531667/
https://www.ncbi.nlm.nih.gov/pubmed/34712574
http://dx.doi.org/10.1016/j.ymgmr.2021.100811