title: Cell-based therapies for Huntington's disease.
abstract: Cell-based therapies are a viable option for the long-term treatment of Huntington's disease (HD), which is characterized by progressive neurodegeneration predominately in the striatum and cortex. Current research focuses on genetic suppression of the mutant huntingtin (mHTT) gene and cell replacement therapy of the lost cells in HD. As we discuss here, the recent development of induced pluripotent stem (iPS) cells technology demonstrated the potential of cell-based therapy in rodent models. It was shown that iPSCs were capable of differentiating into lost neurons in HD and stem cell grafts can improve motor deficiency in HD rodent models. Altogether, these findings have shown great promise for developing the foundation of the cell-based therapy.
authors.affiliation: Division of Neuropharmacology and Neurologic Diseases, Yerkes National Primate Research Center, 954 Gatewood Rd, NE Atlanta, GA 30329, USA; Department of Human Genetics, Emory University School of Medicine, 615 Michael St, Atlanta, GA 30322, USA.
authors.forename: Yiju
authors.initials: Y
authors.lastname: Chen
authors.affiliation: Division of Neuropharmacology and Neurologic Diseases, Yerkes National Primate Research Center, 954 Gatewood Rd, NE Atlanta, GA 30329, USA; Department of Human Genetics, Emory University School of Medicine, 615 Michael St, Atlanta, GA 30322, USA; Genetics and Molecular Biology Program, Laney Graduate School, Emory University, 201 Dowman Drive, Atlanta, GA 30322, USA.
authors.forename: Richard L
authors.initials: RL
authors.lastname: Carter
authors.affiliation: Division of Neuropharmacology and Neurologic Diseases, Yerkes National Primate Research Center, 954 Gatewood Rd, NE Atlanta, GA 30329, USA; Department of Human Genetics, Emory University School of Medicine, 615 Michael St, Atlanta, GA 30322, USA; Genetics and Molecular Biology Program, Laney Graduate School, Emory University, 201 Dowman Drive, Atlanta, GA 30322, USA.
authors.forename: In K
authors.initials: IK
authors.lastname: Cho
authors.affiliation: Division of Neuropharmacology and Neurologic Diseases, Yerkes National Primate Research Center, 954 Gatewood Rd, NE Atlanta, GA 30329, USA; Department of Human Genetics, Emory University School of Medicine, 615 Michael St, Atlanta, GA 30322, USA; Genetics and Molecular Biology Program, Laney Graduate School, Emory University, 201 Dowman Drive, Atlanta, GA 30322, USA. Electronic address: awchan@emory.edu.
authors.forename: Anthony W S
authors.initials: AW
authors.lastname: Chan
mesh_terms: D000818:Animals; D064987:Cell- and Tissue-Based Therapy / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D000071058:Huntingtin Protein; D006816:Huntington Disease / Q000175:diagnosis / Q000235:genetics* / Q000628:therapy*; D057026:Induced Pluripotent Stem Cells / Q000637:transplantation; D009419:Nerve Tissue Proteins / Q000037:antagonists & inhibitors / Q000235:genetics
chemical_list: C086055:HTT protein, human; D000071058:Huntingtin Protein; D009419:Nerve Tissue Proteins