Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses
Summary
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.
| Authors | Sima N, Li R, Huang W, Xu M, Beers J, Zou J, Titus S, Ottinger EA, Marugan JJ, Xie X, Zheng W |
|---|---|
| Journal | Orphanet journal of rare diseases |
| Publication Date | 2018 Apr 10;13(1):54 |
| PubMed | 29631617 |
| PubMed Central | PMC5891977 |
| DOI | 10.1186/s13023-018-0798-2 |