In Vivo Roles of a Patient-Derived Induced Pluripotent Stem Cell Line (HD72-iPSC) in the YAC128 Model of Huntington's Disease.
- Author:
Iksoo JEON
1
;
Chunggab CHOI
;
Nayeon LEE
;
Wooseok IM
;
Manho KIM
;
Seung Hun OH
;
In Hyun PARK
;
Hyun Sook KIM
;
Jihwan SONG
Author Information
1. CHA Stem Cell Institute, CHA University, Seoul, Korea. jsong@cha.ac.kr
- Publication Type:Brief Communication
- Keywords:
Huntington's disease (HD);
Induced pluripotent stem cells (iPSCs);
YAC128 transgenic mice;
GABAergic neurons;
Aggregate formation
- MeSH:
Animals;
GABAergic Neurons;
Humans;
Huntington Disease*;
Induced Pluripotent Stem Cells;
Mice;
Mice, Transgenic;
Neurons;
Pathology;
Pluripotent Stem Cells*;
Transplants
- From:International Journal of Stem Cells
2014;7(1):43-47
- CountryRepublic of Korea
- Language:English
-
Abstract:
Induced pluripotent stem cells (iPSCs) generated from somatic cells of patients can provide immense opportunities to model human diseases, which may lead to develop novel therapeutics. Huntington's disease (HD) is a devastating neurodegenerative genetic disease, with no available therapeutic options at the moment. We recently reported the characteristics of a HD patient-derived iPSC carrying 72 CAG repeats (HD72-iPSC). In this study, we investigated the in vivo roles of HD72-iPSC in the YAC128 transgenic mice, a commonly used HD mouse model carrying 128 CAG repeats. To do this, we transplanted HD72-iPSC-derived neural precursors into the striatum of YAC128 mice bilaterally and observed a significant behavioral improvement in the grafted mice. Interestingly, the transplanted HD72-iPSC-derived neural precursors formed GABAeric neurons efficiently, but no EM48-positive protein aggregates were detected at 12 weeks after transplantation. Taken together, these results indicate no HD pathology was developed from the grafted cells, or no transmission of HD pathology from the host to the graft occurred at 12 weeks post-transplantation.
