title: The expanding world of stem cell modeling of Huntington's disease: creating tools with a promising future.
abstract: Deconstructing the mechanistic basis of neurodegenerative disorders, such as Huntington's disease (HD), has been a particularly challenging undertaking, relying mostly on post-mortem tissue samples, non-neural cell lines from affected individuals, and model organisms. Two articles recently published in Cell Stem Cell report first the generation and characterization of induced pluripotent stem cell (iPSC)-derived models for HD, and second, the genetic correction of a disease-causing CAG expansion mutation in iPSCs from individuals with HD. Taken together, these two studies provide a framework for the production and validation of iPSC materials for human neurodegenerative disease research and yield crucial tools for investigating future therapies.
authors.affiliation: Department of Pediatrics, University of California, San Diego, 9500 Gilman Drive, MC 0642, La Jolla, CA 92037, USA ; Institute for Genomic Medicine, University of California, San Diego, 9500 Gilman Drive, MC 0642, La Jolla, CA 92037, USA ; Sanford Consortium for Regenerative Medicine, University of California, San Diego, 9500 Gilman Drive, MC 0642, La Jolla, CA 92037, USA.
authors.forename: Jacqueline M
authors.initials: JM
authors.lastname: Ward
authors.affiliation: Department of Pediatrics, University of California, San Diego, 9500 Gilman Drive, MC 0642, La Jolla, CA 92037, USA ; Institute for Genomic Medicine, University of California, San Diego, 9500 Gilman Drive, MC 0642, La Jolla, CA 92037, USA ; Sanford Consortium for Regenerative Medicine, University of California, San Diego, 9500 Gilman Drive, MC 0642, La Jolla, CA 92037, USA ; Department of Cellular and Molecular Medicine, University of California, San Diego, 9500 Gilman Drive, MC 0642, La Jolla, CA 92037, USA ; Department of Neurosciences, University of California, San Diego, 9500 Gilman Drive, MC 0642, La Jolla, CA 92037, USA ; Division of Biological Sciences, University of California, San Diego, 9500 Gilman Drive, MC 0642, La Jolla, CA 92037, USA ; Rady Children's Hospital, San Diego, CA 92193, USA.
authors.forename: Albert R
authors.initials: AR
authors.lastname: La Spada