Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses

BACKGROUND: Infantile and late infantile neuronal ceroid lipofuscinoses (NCLs) are lysosomal storage diseases affecting the central nervous system (CNS). The infantile NCL (INCL) is caused by mutations in the PPT1 gene and late-infantile NCL (LINCL) is due to mutations in the TPP1 gene. Deficiency i...

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Detalles Bibliográficos
Autores principales: Sima, Ni, Li, Rong, Huang, Wei, Xu, Miao, Beers, Jeanette, Zou, Jizhong, Titus, Steven, Ottinger, Elizabeth A., Marugan, Juan J., Xie, Xing, Zheng, Wei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5891977/
https://www.ncbi.nlm.nih.gov/pubmed/29631617
http://dx.doi.org/10.1186/s13023-018-0798-2

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