Cell-based therapies for Huntington's disease

• Type of Material: Article
• Authors: Yiju Chen, Emory University; Richard L. Carter, Emory University; In Ki Cho, Emory University; Anthony Chan, Emory University
• Language: English
• Date: 2014-07-01
• Publisher: Elsevier: 12 months
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2014 Elsevier Ltd.
• License: Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1016/j.drudis.2014.02.012
• Title of Journal or Parent Work: Drug Discovery Today
• ISSN: 1359-6446
• Volume: 19
• Issue: 7
• Start Page: 980
• End Page: 984
• Grant/Funding Information: Yerkes National Primate Research Center is supported by the National Center for Research Resources P51RR165 and is currently supported by the Office of Research and Infrastructure Program (ORIP)/OD P51OD11132.; A.W.S.C. is supported by the National Institutes of Health (OD010930; NS064991; MH100670; NS084163).
• Keywords: MUTANT HUNTINGTIN; STRIATAL ALLOGRAFTS; NEURONS; GENE-EXPRESSION; IN-VIVO TRACKING; ALLELE-SELECTIVE INHIBITION; Science & Technology; Life Sciences & Biomedicine; MAGNETIC NANOPARTICLES; MOUSE; Pharmacology & Pharmacy; FETAL NEURAL TRANSPLANTS; ANTISENSE OLIGONUCLEOTIDES
• Research Categories: Biology, Cell; Biology, Neuroscience; Biology, Genetics

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.