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Neural stem cells for disease modeling of Wolman disease and evaluation of therapeutics

BACKGROUND: Wolman disease (WD) is a rare lysosomal storage disorder that is caused by mutations in the LIPA gene encoding lysosomal acid lipase (LAL). Deficiency in LAL function causes accumulation of cholesteryl esters and triglycerides in lysosomes. Fatality usually occurs within the first year o...

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Detalles Bibliográficos
Autores principales:	Aguisanda, Francis, Yeh, Charles D., Chen, Catherine Z., Li, Rong, Beers, Jeanette, Zou, Jizhong, Thorne, Natasha, Zheng, Wei
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2017
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5490176/ https://www.ncbi.nlm.nih.gov/pubmed/28659158 http://dx.doi.org/10.1186/s13023-017-0670-9

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