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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...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2021
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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 |