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

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作者：
Ni Sima
Rong Li
Wei Huang
Miao Xu
Jeanette Beers
Jizhong Zou
Steven Titus
Elizabeth A. Ottinger
Juan J. Marugan
Xing Xie
Wei Zheng
机构：
[1] National Institutes of Health,National Center for Advancing Translational Sciences
[2] Women’s Reproductive Health Laboratory of Zhejiang Province,Department of Gynecologic Oncology
[3] Women’s Hospital,iPSC core, National Heart
[4] School of Medicine,undefined
[5] Zhejiang University,undefined
[6] Lung and Blood Institute,undefined
[7] National Institutes of Health,undefined
来源：
Orphanet Journal of Rare Diseases | / 13卷
关键词：
Neuronal ceroid lipofuscinosis; INCL; LINCL; Lysosomal storage disease; Induced pluripotent stem cells; Neural stem cells; Enzyme replacement therapy; Cyclodextrin; δ-tocopherol;
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