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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Detalhes bibliográficos
Publicado no:Stem Cells Transl Med
Principais autores: 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: Artigo
Idioma:Inglês
Publicado em: AlphaMed Press 2016
Assuntos:
Cell-Based Drug Development, Screening, and Toxicology
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5189647/
https://ncbi.nlm.nih.gov/pubmed/27484861
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.5966/sctm.2015-0373
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