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A novel CRISPR/Cas9-based iduronate-2-sulfatase (IDS) knockout human neuronal cell line reveals earliest pathological changes

Multiple complex intracellular cascades contributing to Hunter syndrome (mucopolysaccharidosis type II) pathogenesis have been recognized and documented in the past years. However, the hierarchy of early cellular abnormalities leading to irreversible neuronal damage is far from being completely unde...

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Detalles Bibliográficos
Autores principales: Badenetti, Lorenzo, Manzoli, Rosa, Trevisan, Marta, D’Avanzo, Francesca, Tomanin, Rosella, Moro, Enrico
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10290981/
https://www.ncbi.nlm.nih.gov/pubmed/37357221
http://dx.doi.org/10.1038/s41598-023-37138-5