Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses.
Ni SimaRong LiWei HuangMiao XuJeanette BeersJizhong ZouSteven TitusElizabeth A OttingerJuan J MaruganXing XieWei ZhengPublished in: Orphanet journal of rare diseases (2018)
The results demonstrate that these patient iPSC derived NCL NSCs are valid cell- based disease models with characteristic disease phenotypes that can be used for study of disease pathophysiology and drug development.