Neuronal and Astrocytic Differentiation from Sanfilippo C Syndrome iPSCs for Disease Modeling and Drug Development

Neuronal and Astrocytic Differentiation from Sanfilippo C Syndrome iPSCs for Disease Modeling and Drug Development

Sanfilippo syndrome type C (mucopolysaccharidosis IIIC) is an early-onset neurodegenerative lysosomal storage disorder, which is currently untreatable. The vast majority of studies focusing on disease mechanisms of Sanfilippo syndrome were performed on non-neural cells or mouse models, which present...

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Bibliographic Details
Main Authors: Noelia Benetó, Monica Cozar, Laura Castilla-Vallmanya, Oskar G. Zetterdahl, Madalina Sacultanu, Eulalia Segur-Bailach, María García-Morant, Antonia Ribes, Henrik Ahlenius, Daniel Grinberg, Lluïsa Vilageliu, Isaac Canals
Format: Article
Language:English
Published: MDPI AG 2020-02-01
Series:Journal of Clinical Medicine
Subjects:
sanfilippo syndrome
mucopolysaccharidosis iii
lysosomal storage disorders
induced pluripotent stem cells
neuronal differentiation
astrocyte differentiation
transcription factor-based differentiation
lysosomes
sirnas
substrate reduction therapy
Online Access:https://www.mdpi.com/2077-0383/9/3/644

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https://www.mdpi.com/2077-0383/9/3/644

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