title: In Vivo Roles of a Patient-Derived Induced Pluripotent Stem Cell Line (HD72-iPSC) in the YAC128 Model of Huntington's Disease.
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.
authors.affiliation: CHA Stem Cell Institute, CHA University.
authors.forename: Iksoo
authors.initials: I
authors.lastname: Jeon
authors.affiliation: CHA Stem Cell Institute, CHA University.
authors.forename: Chunggab
authors.initials: C
authors.lastname: Choi
authors.affiliation: CHA Stem Cell Institute, CHA University.
authors.forename: Nayeon
authors.initials: N
authors.lastname: Lee
authors.affiliation: Department of Neurology, Biomedical Research Institute, Seoul National University Hospital, Seoul.
authors.forename: Wooseok
authors.initials: W
authors.lastname: Im
authors.affiliation: Department of Neurology, Biomedical Research Institute, Seoul National University Hospital, Seoul.
authors.forename: Manho
authors.initials: M
authors.lastname: Kim
authors.affiliation: Department of Neurology, CHA Bundang Medical Center, CHA University, Seongnam, Korea.
authors.forename: Seung-Hun
authors.initials: SH
authors.lastname: Oh
authors.affiliation: Department of Genetics, Yale University School of Medicine, New Haven, USA.
authors.forename: In-Hyun
authors.initials: IH
authors.lastname: Park
authors.affiliation: Department of Neurology, CHA Bundang Medical Center, CHA University, Seongnam, Korea.
authors.forename: Hyun Sook
authors.initials: HS
authors.lastname: Kim
authors.affiliation: CHA Stem Cell Institute, CHA University.
authors.forename: Jihwan
authors.initials: J
authors.lastname: Song
keywords: Aggregate formation; GABAergic neurons; Huntington’s disease (HD); Induced pluripotent stem cells (iPSCs); YAC128 transgenic mice