Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient- and cell type-specific disease phenotypes

Globoid cell leukodystrophy (GLD) is a rare neurodegenerative lysosomal storage disease caused by an inherited deficiency of β-galactocerebrosidase (GALC). GLD pathogenesis and therapeutic correction have been poorly studied in patient neural cells. Here, we investigated the impact of GALC deficienc...

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Publicat a:Stem Cell Reports
Autors principals: Mangiameli, Elisabeth, Cecchele, Anna, Morena, Francesco, Sanvito, Francesca, Matafora, Vittoria, Cattaneo, Angela, della Volpe, Lucrezia, Gnani, Daniela, Paulis, Marianna, Susani, Lucia, Martino, Sabata, Di Micco, Raffaella, Bachi, Angela, Gritti, Angela
Format: Artigo
Idioma:Inglês
Publicat: Elsevier 2021
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC8190599/
https://ncbi.nlm.nih.gov/pubmed/33989519
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2021.04.011
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