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Induced Pluripotent Stem Cell Modeling of Gaucher’s Disease: What Have We Learned?

Gaucher’s disease (GD) is the most frequently inherited lysosomal storage disease, presenting both visceral and neurologic symptoms. Mutations in acid β-glucocerebrosidase disrupt the sphingolipid catabolic pathway promoting glucosylceramide (GlcCer) accumulation in lysosomes. Current treatment opti...

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Detalles Bibliográficos
Autores principales:	Santos, Dino Matias, Tiscornia, Gustavo
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2017
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5412467/ https://www.ncbi.nlm.nih.gov/pubmed/28430167 http://dx.doi.org/10.3390/ijms18040888

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