Manho Kim1 Wooseok Im1 and Tian Liu2
Posters-Accepted Abstracts: J Stem Cell Res Ther
Huntington’s disease (HD) is a neurodegenerative disease caused by a CAG expansion in the gene encoding huntingtin
(HTT). HD presents with cognitive deficit with chorea by neuronal dysfunction due to loss of cortical and striatal neurons.
The decreased of microRNA-124 level both in the brains of transgenic mice and in human has been reported. MicroRNA-124
contributes to neurogenesis through regulating its targets. However, whether microRNA-124 has potential to improve the
HD has not been investigated. Transgenic HD mice of R6/2 line (B6CBA-Tg (HDexon1) 62Gpb/3J, 111 CAGs) and their
wild type littermates were used for the efficacy of miRNA-124 delivery. 20 nmol of MicroRNA-124 injection into bilateral
striatum was performed on 8 weeks-old mice. Rota-rod test monitoring and BrdU staining were performed. MicroRNA-124
increased the latency to fall of R6/2 HD transgenic mice on the rotarod test. BrdU staining in the sub ventricular zone shows
the increase of neurogenesis. In addition, BDNF (Brain-derived neurotrophic factor) and PGC1α protein levels in striatum
were increased whereas SOX9 was decreased. This result suggests that delivery of microRNA-124 in the HD brain increases
neuronal differentiation which leads to slow the progression in HD.
Manho Kim has completed his MD (1990), PhD (2002) from from Seoul National University School of Medicine with Postdoc experience in MGH Neurology (1996-
1999). He is a Professor in Neurology, Seoul National University Hospital, with academic interest in the Huntington’s disease and other neurodegenerative disorder.
In vitro, in vivo model and HD patients enabled him to applying translational approach, recently focused on the stem cell treatment and miRNA based therapeutic
strategy. He has been in the editorial board of Korean Society for Stem Cell Research (IJST) with more than 200 papers published in reputed journals.