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Induced Pluripotent Stem Cells for Disease Modeling and Evaluation of Therapeutics for Niemann-Pick Disease Type A

Niemann-Pick disease type A (NPA) is a lysosomal storage disease caused by mutations in the SMPD1 gene that encodes acid sphingomyelinase (ASM). Deficiency in ASM function results in lysosomal accumulation of sphingomyelin and neurodegeneration. Currently, there is no effective treatment for NPA. To...

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Detalles Bibliográficos
Autores principales:	Long, Yan, Xu, Miao, Li, Rong, Dai, Sheng, Beers, Jeanette, Chen, Guokai, Soheilian, Ferri, Baxa, Ulrich, Wang, Mengqiao, Marugan, Juan J., Muro, Silvia, Li, Zhiyuan, Brady, Roscoe, Zheng, Wei
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	AlphaMed Press 2016
Materias:	Cell-Based Drug Development, Screening, and Toxicology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5189647/ https://www.ncbi.nlm.nih.gov/pubmed/27484861 http://dx.doi.org/10.5966/sctm.2015-0373

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