{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,13]],"date-time":"2026-07-13T16:39:53Z","timestamp":1783960793597,"version":"3.55.0"},"reference-count":215,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2019,10,1]],"date-time":"2019-10-01T00:00:00Z","timestamp":1569888000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2019,10,1]],"date-time":"2019-10-01T00:00:00Z","timestamp":1569888000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,8,22]],"date-time":"2023-08-22T00:00:00Z","timestamp":1692662400000},"content-version":"vor","delay-in-days":1421,"URL":"http:\/\/www.elsevier.com\/open-access\/userlicense\/1.0\/"}],"funder":[{"DOI":"10.13039\/100000065","name":"National Institute of Neurological Disorders and Stroke","doi-asserted-by":"publisher","award":["NS100529"],"award-info":[{"award-number":["NS100529"]}],"id":[{"id":"10.13039\/100000065","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000065","name":"National Institute of Neurological Disorders and Stroke","doi-asserted-by":"publisher","award":["NS094422"],"award-info":[{"award-number":["NS094422"]}],"id":[{"id":"10.13039\/100000065","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000049","name":"National Institute on Aging","doi-asserted-by":"publisher","award":["AG000266"],"award-info":[{"award-number":["AG000266"]}],"id":[{"id":"10.13039\/100000049","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["neurotherapeuticsjournal.org","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Neurotherapeutics"],"published-print":{"date-parts":[[2019,10]]},"DOI":"10.1007\/s13311-019-00810-8","type":"journal-article","created":{"date-parts":[[2019,12,2]],"date-time":"2019-12-02T13:51:29Z","timestamp":1575294689000},"page":"979-998","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":26,"title":["Modeling Polyglutamine Expansion Diseases with Induced Pluripotent Stem Cells"],"prefix":"10.1016","volume":"16","author":[{"given":"Swati","family":"Naphade","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kizito-Tshitoko","family":"Tshilenge","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9050-7977","authenticated-orcid":false,"given":"Lisa M.","family":"Ellerby","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"7147","key":"10.1007\/s13311-019-00810-8_bib1","doi-asserted-by":"crossref","first-page":"932","DOI":"10.1038\/nature05977","article-title":"Expandable DNA repeats and human disease","volume":"447","author":"Mirkin","year":"2007","journal-title":"Nature."},{"issue":"11","key":"10.1007\/s13311-019-00810-8_bib2","doi-asserted-by":"crossref","first-page":"786","DOI":"10.1038\/nrg2828","article-title":"Mechanisms of trinucleotide repeat instability during human development","volume":"11","author":"McMurray","year":"2010","journal-title":"Nat Rev Genet."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib3","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1038\/nrg2748","article-title":"Repeat expansion disease: progress and puzzles in disease pathogenesis","volume":"11","author":"La Spada","year":"2010","journal-title":"Nat Rev Genet."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib4","doi-asserted-by":"crossref","first-page":"453","DOI":"10.1111\/j.1440-1789.2010.01120.x","article-title":"Dentatorubral-pallidoluysian atrophy (DRPLA): The 50th Anniversary of Japanese Society of Neuropathology","volume":"30","author":"Yamada","year":"2010","journal-title":"Neuropathology."},{"key":"10.1007\/s13311-019-00810-8_bib5","doi-asserted-by":"crossref","first-page":"577","DOI":"10.5334\/tohm.439","article-title":"Dentatorubral-pallidoluysian Atrophy: An Update","volume":"8","author":"Carroll","year":"2018","journal-title":"Tremor Other Hyperkinet Mov (N Y)"},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib6","doi-asserted-by":"crossref","first-page":"971","DOI":"10.1016\/0092-8674(93)90585-E","article-title":"A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. The Huntington's Disease Collaborative Research Group.","volume":"72","year":"1993","journal-title":"Cell."},{"issue":"3","key":"10.1007\/s13311-019-00810-8_bib7","doi-asserted-by":"crossref","first-page":"905","DOI":"10.1152\/physrev.00041.2009","article-title":"Molecular mechanisms and potential therapeutical targets in Huntington's disease","volume":"90","author":"Zuccato","year":"2010","journal-title":"Physiol Rev."},{"issue":"6330","key":"10.1007\/s13311-019-00810-8_bib8","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1038\/352077a0","article-title":"Androgen receptor gene mutations in X-linked spinal and bulbar muscular atrophy","volume":"352","author":"La Spada","year":"1991","journal-title":"Nature."},{"key":"10.1007\/s13311-019-00810-8_bib9","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1007\/978-3-319-71779-1_5","article-title":"X-Linked Spinal and Bulbar Muscular Atrophy: From Clinical Genetic Features and Molecular Pathology to Mechanisms Underlying Disease Toxicity","volume":"1049","author":"Cortes","year":"2018","journal-title":"Adv Exp Med Biol."},{"key":"10.1007\/s13311-019-00810-8_bib10","doi-asserted-by":"crossref","DOI":"10.1007\/s13311-018-00696-y","article-title":"Genetics, Mechanisms, and Therapeutic Progress in Polyglutamine Spinocerebellar Ataxias.","author":"Buijsen","year":"2019","journal-title":"Neurotherapeutics."},{"issue":"10","key":"10.1007\/s13311-019-00810-8_bib11","doi-asserted-by":"crossref","first-page":"590","DOI":"10.1038\/s41582-018-0051-6","article-title":"Spinocerebellar ataxias: prospects and challenges for therapy development","volume":"14","author":"Ashizawa","year":"2018","journal-title":"Nat Rev Neurol."},{"issue":"3","key":"10.1007\/s13311-019-00810-8_bib12","doi-asserted-by":"crossref","first-page":"417","DOI":"10.1016\/j.scr.2013.01.009","article-title":"EZ spheres: a stable and expandable culture system for the generation of pre-rosette multipotent stem cells from human ESCs and iPSCs","volume":"10","author":"Ebert","year":"2013","journal-title":"Stem Cell Res."},{"issue":"7","key":"10.1007\/s13311-019-00810-8_bib13","doi-asserted-by":"crossref","first-page":"C520","DOI":"10.1152\/ajpcell.00166.2015","article-title":"Forced cell cycle exit and modulation of GABAA, CREB, and GSK3beta signaling promote functional maturation of induced pluripotent stem cell-derived neurons","volume":"310","author":"Telezhkin","year":"2016","journal-title":"Am J Physiol Cell Physiol."},{"issue":"7378","key":"10.1007\/s13311-019-00810-8_bib14","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1038\/nature10648","article-title":"Dopamine neurons derived from human ES cells efficiently engraft in animal models of Parkinson's disease","volume":"480","author":"Kriks","year":"2011","journal-title":"Nature."},{"issue":"43","key":"10.1007\/s13311-019-00810-8_bib15","doi-asserted-by":"crossref","first-page":"16707","DOI":"10.1073\/pnas.0808488105","article-title":"Striatal progenitors derived from human ES cells mature into DARPP32 neurons in vitro and in quinolinic acid-lesioned rats","volume":"105","author":"Aubry","year":"2008","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"7","key":"10.1007\/s13311-019-00810-8_bib16","doi-asserted-by":"crossref","first-page":"e11853","DOI":"10.1371\/journal.pone.0011853","article-title":"Specification of region-specific neurons including forebrain glutamatergic neurons from human induced pluripotent stem cells","volume":"5","author":"Zeng","year":"2010","journal-title":"PLoS One."},{"issue":"24","key":"10.1007\/s13311-019-00810-8_bib17","doi-asserted-by":"crossref","first-page":"3995","DOI":"10.1007\/s00018-011-0770-y","article-title":"Specification of neuronal and glial subtypes from human pluripotent stem cells","volume":"68","author":"Liu","year":"2011","journal-title":"Cell Mol Life Sci."},{"issue":"7384","key":"10.1007\/s13311-019-00810-8_bib18","doi-asserted-by":"crossref","first-page":"216","DOI":"10.1038\/nature10821","article-title":"Probing sporadic and familial Alzheimer's disease using induced pluripotent stem cells","volume":"482","author":"Israel","year":"2012","journal-title":"Nature."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib19","doi-asserted-by":"crossref","first-page":"487","DOI":"10.1016\/j.stem.2013.01.009","article-title":"Modeling Alzheimer's disease with iPSCs reveals stress phenotypes associated with intracellular Abeta and differential drug responsiveness","volume":"12","author":"Kondo","year":"2013","journal-title":"Cell Stem Cell."},{"issue":"15","key":"10.1007\/s13311-019-00810-8_bib20","doi-asserted-by":"crossref","first-page":"5803","DOI":"10.1073\/pnas.1202922109","article-title":"Mutant induced pluripotent stem cell lines recapitulate aspects of TDP-43 proteinopathies and reveal cell-specific vulnerability","volume":"109","author":"Bilican","year":"2012","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib21","doi-asserted-by":"crossref","first-page":"380","DOI":"10.1002\/emmm.201200215","article-title":"Disease-specific phenotypes in dopamine neurons from human iPS-based models of genetic and sporadic Parkinson's disease","volume":"4","author":"Sanchez-Danes","year":"2012","journal-title":"EMBO Mol Med."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib22","doi-asserted-by":"crossref","first-page":"897","DOI":"10.1016\/j.cell.2018.02.011","article-title":"Dissecting the Causal Mechanism of X-Linked Dystonia-Parkinsonism by Integrating Genome and Transcriptome Assembly","volume":"172","author":"Aneichyk","year":"2018","journal-title":"Cell."},{"key":"10.1007\/s13311-019-00810-8_bib23","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1016\/B978-0-444-63233-3.00011-7","article-title":"The CAG-polyglutamine repeat diseases: a clinical, molecular, genetic, and pathophysiologic nosology","volume":"147","author":"Stoyas","year":"2018","journal-title":"Handb Clin Neurol."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib24","doi-asserted-by":"crossref","first-page":"1147","DOI":"10.1016\/S0896-6273(00)80405-5","article-title":"Intranuclear neuronal inclusions: a common pathogenic mechanism for glutamine-repeat neurodegenerative diseases?","volume":"19","author":"Ross","year":"1997","journal-title":"Neuron."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib25","doi-asserted-by":"crossref","first-page":"275","DOI":"10.1016\/S0896-6273(00)80839-9","article-title":"Nuclear accumulation of truncated atrophin-1 fragments in a transgenic mouse model of DRPLA","volume":"24","author":"Schilling","year":"1999","journal-title":"Neuron."},{"issue":"5334","key":"10.1007\/s13311-019-00810-8_bib26","doi-asserted-by":"crossref","first-page":"1990","DOI":"10.1126\/science.277.5334.1990","article-title":"Aggregation of huntingtin in neuronal intranuclear inclusions and dystrophic neurites in brain","volume":"277","author":"DiFiglia","year":"1997","journal-title":"Science."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib27","doi-asserted-by":"crossref","first-page":"339","DOI":"10.1086\/302269","article-title":"Protein fate in neurodegenerative proteinopathies: polyglutamine diseases join the (mis)fold","volume":"64","author":"Paulson","year":"1999","journal-title":"Am J Hum Genet."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib28","doi-asserted-by":"crossref","first-page":"416","DOI":"10.1016\/j.yfrne.2011.06.003","article-title":"Neurotoxic effects of androgens in spinal and bulbar muscular atrophy","volume":"32","author":"Parodi","year":"2011","journal-title":"Front Neuroendocrinol."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib29","doi-asserted-by":"crossref","first-page":"843","DOI":"10.1016\/S0896-6273(02)00834-6","article-title":"Testosterone reduction prevents phenotypic expression in a transgenic mouse model of spinal and bulbar muscular atrophy","volume":"35","author":"Katsuno","year":"2002","journal-title":"Neuron."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib30","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1093\/hmg\/8.5.731","article-title":"Polyglutamine-expanded androgen receptors form aggregates that sequester heat shock proteins, proteasome components and SRC-1, and are suppressed by the HDJ-2 chaperone","volume":"8","author":"Stenoien","year":"1999","journal-title":"Hum Mol Genet."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib31","doi-asserted-by":"crossref","first-page":"119","DOI":"10.1038\/s41598-018-36784-4","article-title":"Impaired Nuclear Export of Polyglutamine-Expanded Androgen Receptor in Spinal and Bulbar Muscular Atrophy","volume":"9","author":"Arnold","year":"2019","journal-title":"Sci Rep."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib32","doi-asserted-by":"crossref","first-page":"106","DOI":"10.1007\/s12311-008-0009-0","article-title":"Clinical, genetic, molecular, and pathophysiological insights into spinocerebellar ataxia type 1","volume":"7","author":"Matilla-Duenas","year":"2008","journal-title":"Cerebellum."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib33","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1016\/S0092-8674(00)81781-X","article-title":"Ataxin-1 nuclear localization and aggregation: role in polyglutamine-induced disease in SCA1 transgenic mice","volume":"95","author":"Klement","year":"1998","journal-title":"Cell."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib34","doi-asserted-by":"crossref","first-page":"3648","DOI":"10.1038\/s41467-018-06041-3","article-title":"Staufen1 links RNA stress granules and autophagy in a model of neurodegeneration","volume":"9","author":"Paul","year":"2018","journal-title":"Nat Commun."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib35","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1038\/79162","article-title":"Nuclear localization or inclusion body formation of ataxin-2 are not necessary for SCA2 pathogenesis in mouse or human","volume":"26","author":"Huynh","year":"2000","journal-title":"Nat Genet."},{"key":"10.1007\/s13311-019-00810-8_bib36","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1146\/annurev-pathmechdis-012418-012857","article-title":"Polyglutamine Repeats in Neurodegenerative Diseases","volume":"14","author":"Lieberman","year":"2019","journal-title":"Annu Rev Pathol."},{"issue":"10","key":"10.1007\/s13311-019-00810-8_bib37","doi-asserted-by":"crossref","first-page":"743","DOI":"10.1038\/nrg1691","article-title":"Diseases of unstable repeat expansion: mechanisms and common principles","volume":"6","author":"Gatchel","year":"2005","journal-title":"Nat Rev Genet."},{"issue":"7154","key":"10.1007\/s13311-019-00810-8_bib38","doi-asserted-by":"crossref","first-page":"704","DOI":"10.1038\/nature06022","article-title":"Global changes to the ubiquitin system in Huntington's disease","volume":"448","author":"Bennett","year":"2007","journal-title":"Nature."},{"issue":"7146","key":"10.1007\/s13311-019-00810-8_bib39","doi-asserted-by":"crossref","first-page":"859","DOI":"10.1038\/nature05853","article-title":"HDAC6 rescues neurodegeneration and provides an essential link between autophagy and the UPS","volume":"447","author":"Pandey","year":"2007","journal-title":"Nature."},{"key":"10.1007\/s13311-019-00810-8_bib40","doi-asserted-by":"crossref","DOI":"10.1007\/s13311-019-00782-9","article-title":"Cell-Autonomous and Non-cell-Autonomous Pathogenic Mechanisms in Huntington's Disease: Insights from In Vitro and In Vivo Models","author":"Creus-Muncunill","year":"2019","journal-title":"Neurotherapeutics."},{"key":"10.1007\/s13311-019-00810-8_bib41","doi-asserted-by":"crossref","DOI":"10.1007\/s13311-019-00777-6","article-title":"Molecular Mechanisms and Therapeutics for Spinocerebellar Ataxia Type 2","author":"Egorova","year":"2019","journal-title":"Neurotherapeutics."},{"key":"10.1007\/s13311-019-00810-8_bib42","doi-asserted-by":"crossref","DOI":"10.1007\/s13311-019-00764-x","article-title":"Molecular Mechanisms and Therapeutics for the GAA.TTC Expansion Disease Friedreich Ataxia","author":"Gottesfeld","year":"2019","journal-title":"Neurotherapeutics."},{"key":"10.1007\/s13311-019-00810-8_bib43","doi-asserted-by":"crossref","DOI":"10.1007\/s13311-019-00762-z","article-title":"Molecular Mechanisms and Therapeutics for SCA17","author":"Liu","year":"2019","journal-title":"Neurotherapeutics."},{"key":"10.1007\/s13311-019-00810-8_bib44","doi-asserted-by":"crossref","DOI":"10.1007\/s13311-019-00768-7","article-title":"DNA Damage Repair in Huntington's Disease and Other Neurodegenerative Diseases.","author":"Maiuri","year":"2019","journal-title":"Neurotherapeutics."},{"key":"10.1007\/s13311-019-00810-8_bib45","doi-asserted-by":"crossref","DOI":"10.1007\/s13311-019-00778-5","article-title":"Molecular Targets and Therapeutic Strategies in Spinocerebellar Ataxia Type 7","author":"Niewiadomska-Cimicka","year":"2019","journal-title":"Neurotherapeutics."},{"key":"10.1007\/s13311-019-00810-8_bib46","doi-asserted-by":"crossref","DOI":"10.1007\/s13311-019-00763-y","article-title":"Moving Towards Therapy in SCA1: Insights from Molecular Mechanisms, Identification of Novel Targets, and Planning for Human Trials","author":"Srinivasan","year":"2019","journal-title":"Neurotherapeutics."},{"key":"10.1007\/s13311-019-00810-8_bib47","doi-asserted-by":"crossref","first-page":"101551","DOI":"10.1016\/j.scr.2019.101551","article-title":"Generation of induced pluripotent stem cell line CSSi008-A (4698) from a patient affected by advanced stage of Dentato-Rubral-Pallidoluysian atrophy (DRPLA)","volume":"40","author":"Bidollari","year":"2019","journal-title":"Stem Cell Res."},{"key":"10.1007\/s13311-019-00810-8_bib48","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1016\/j.scr.2018.03.018","article-title":"Generation of 3 spinocerebellar ataxia type 1 (SCA1) patient-derived induced pluripotent stem cell lines LUMCi002-A, B, and C and 2 unaffected sibling control induced pluripotent stem cell lines LUMCi003-A and B","volume":"29","author":"Buijsen","year":"2018","journal-title":"Stem Cell Res."},{"key":"10.1007\/s13311-019-00810-8_bib49","doi-asserted-by":"crossref","first-page":"101555","DOI":"10.1016\/j.scr.2019.101555","article-title":"Generation of an induced pluripotent stem cell line (XHCSUi001-A) from urine cells of a patient with spinocerebellar ataxia type 3","volume":"40","author":"He","year":"2019","journal-title":"Stem Cell Res."},{"key":"10.1007\/s13311-019-00810-8_bib50","doi-asserted-by":"crossref","first-page":"101512","DOI":"10.1016\/j.scr.2019.101512","article-title":"Generation of human iPS cell line IBCHi001-A from dentatorubral-pallidoluysian atrophy patient's fibroblasts","volume":"39","author":"Kozlowska","year":"2019","journal-title":"Stem Cell Res."},{"key":"10.1007\/s13311-019-00810-8_bib51","doi-asserted-by":"crossref","first-page":"101361","DOI":"10.1016\/j.scr.2018.101361","article-title":"Generation of Spinocerebellar Ataxia Type 2 induced pluripotent stem cell lines, CHOPi002-A and CHOPi003-A, from patients with abnormal CAG repeats in the coding region of the ATXN2 gene","volume":"34","author":"Maguire","year":"2019","journal-title":"Stem Cell Res."},{"key":"10.1007\/s13311-019-00810-8_bib52","doi-asserted-by":"crossref","first-page":"101498","DOI":"10.1016\/j.scr.2019.101498","article-title":"Generation of 5 induced pluripotent stem cell lines, LUMCi007-A and B and LUMCi008-A, B and C, from 2 patients with Huntington disease","volume":"39","author":"van der Graaf","year":"2019","journal-title":"Stem Cell Res."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib53","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1159\/000443738","article-title":"The Prevalence of Huntington's Disease","volume":"46","author":"Rawlins","year":"2016","journal-title":"Neuroepidemiology."},{"key":"10.1007\/s13311-019-00810-8_bib54","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/978-3-319-71779-1_1","article-title":"Clinical Features of Huntington's Disease","volume":"1049","author":"Ghosh","year":"2018","journal-title":"Adv Exp Med Biol."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib55","doi-asserted-by":"crossref","first-page":"726","DOI":"10.1111\/bpa.12426","article-title":"Huntington's disease (HD): the neuropathology of a multisystem neurodegenerative disorder of the human brain","volume":"26","author":"Rub","year":"2016","journal-title":"Brain Pathol."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib56","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1016\/j.stem.2012.04.026","article-title":"Genetic correction of Huntington's disease phenotypes in induced pluripotent stem cells","volume":"11","author":"An","year":"2012","journal-title":"Cell Stem Cell."},{"key":"10.1007\/s13311-019-00810-8_bib57","doi-asserted-by":"crossref","first-page":"736","DOI":"10.3389\/fnins.2017.00736","article-title":"Altered Expression of Matrix Metalloproteinases and Their Endogenous Inhibitors in a Human Isogenic Stem Cell Model of Huntington's Disease","volume":"11","author":"Naphade","year":"2017","journal-title":"Front Neurosci."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib58","doi-asserted-by":"crossref","first-page":"1023","DOI":"10.1016\/j.stemcr.2015.11.005","article-title":"Genomic Analysis Reveals Disruption of Striatal Neuronal Development and Therapeutic Targets in Human Huntington's Disease Neural Stem Cells","volume":"5","author":"Ring","year":"2015","journal-title":"Stem Cell Reports."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib59","doi-asserted-by":"crossref","first-page":"648","DOI":"10.1038\/nn.4532","article-title":"Developmental alterations in Huntington's disease neural cells and pharmacological rescue in cells and mice","volume":"20","author":"Consortium","year":"2017","journal-title":"Nat Neurosci."},{"key":"10.1007\/s13311-019-00810-8_bib60","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1016\/j.neuint.2017.01.003","article-title":"Characterising the developmental profile of human embryonic stem cell-derived medium spiny neuron progenitors and assessing mature neuron function using a CRISPR-generated human DARPP-32(WT\/eGFP-AMP) reporter line","volume":"106","author":"Hunt","year":"2017","journal-title":"Neurochem Int."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib61","doi-asserted-by":"crossref","first-page":"3351","DOI":"10.1007\/s12035-017-0477-7","article-title":"Huntington Disease as a Neurodevelopmental Disorder and Early Signs of the Disease in Stem Cells","volume":"55","author":"Wiatr","year":"2018","journal-title":"Mol Neurobiol."},{"issue":"5391","key":"10.1007\/s13311-019-00810-8_bib62","doi-asserted-by":"crossref","first-page":"1145","DOI":"10.1126\/science.282.5391.1145","article-title":"Embryonic stem cell lines derived from human blastocysts","volume":"282","author":"Thomson","year":"1998","journal-title":"Science."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib63","doi-asserted-by":"crossref","first-page":"513","DOI":"10.1007\/s12015-011-9290-2","article-title":"GABAergic neurons from mouse embryonic stem cells possess functional properties of striatal neurons in vitro, and develop into striatal neurons in vivo in a mouse model of Huntington's disease","volume":"8","author":"Shin","year":"2012","journal-title":"Stem Cell Rev."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib64","doi-asserted-by":"crossref","first-page":"1820","DOI":"10.1096\/fj.12-219220","article-title":"A novel human embryonic stem cell-derived Huntington's disease neuronal model exhibits mutant huntingtin (mHTT) aggregates and soluble mHTT-dependent neurodegeneration","volume":"27","author":"Lu","year":"2013","journal-title":"FASEB J."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib65","doi-asserted-by":"crossref","first-page":"106","DOI":"10.1016\/S1472-6483(10)60053-3","article-title":"Human embryonic stem cell models of Huntington disease","volume":"19","author":"Niclis","year":"2009","journal-title":"Reprod Biomed Online."},{"key":"10.1007\/s13311-019-00810-8_bib66","doi-asserted-by":"crossref","first-page":"37","DOI":"10.3389\/fncel.2013.00037","article-title":"Characterization of forebrain neurons derived from late-onset Huntington's disease human embryonic stem cell lines","volume":"7","author":"Niclis","year":"2013","journal-title":"Front Cell Neurosci."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib67","doi-asserted-by":"crossref","first-page":"861","DOI":"10.1016\/j.cell.2007.11.019","article-title":"Induction of pluripotent stem cells from adult human fibroblasts by defined factors","volume":"131","author":"Takahashi","year":"2007","journal-title":"Cell."},{"issue":"7151","key":"10.1007\/s13311-019-00810-8_bib68","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1038\/nature05934","article-title":"Generation of germline-competent induced pluripotent stem cells","volume":"448","author":"Okita","year":"2007","journal-title":"Nature."},{"issue":"5858","key":"10.1007\/s13311-019-00810-8_bib69","doi-asserted-by":"crossref","first-page":"1917","DOI":"10.1126\/science.1151526","article-title":"Induced pluripotent stem cell lines derived from human somatic cells","volume":"318","author":"Yu","year":"2007","journal-title":"Science."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib70","doi-asserted-by":"crossref","first-page":"877","DOI":"10.1016\/j.cell.2008.07.041","article-title":"Disease-specific induced pluripotent stem cells","volume":"134","author":"Park","year":"2008","journal-title":"Cell."},{"key":"10.1007\/s13311-019-00810-8_bib71","doi-asserted-by":"crossref","first-page":"RRN1193","DOI":"10.1371\/currents.RRN1193","article-title":"Characterization of Human Huntington's Disease Cell Model from Induced Pluripotent Stem Cells","volume":"2","author":"Zhang","year":"2010","journal-title":"PLoS Curr."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib72","doi-asserted-by":"crossref","first-page":"264","DOI":"10.1016\/j.stem.2012.04.027","article-title":"Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes","volume":"11","author":"Consortium","year":"2012","journal-title":"Cell Stem Cell."},{"issue":"31","key":"10.1007\/s13311-019-00810-8_bib73","doi-asserted-by":"crossref","first-page":"12538","DOI":"10.1073\/pnas.1209979109","article-title":"Donor-dependent variations in hepatic differentiation from human-induced pluripotent stem cells","volume":"109","author":"Kajiwara","year":"2012","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"3","key":"10.1007\/s13311-019-00810-8_bib74","doi-asserted-by":"crossref","first-page":"619","DOI":"10.1016\/j.stemcr.2017.01.022","article-title":"Reversal of Phenotypic Abnormalities by CRISPR\/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells","volume":"8","author":"Xu","year":"2017","journal-title":"Stem Cell Reports."},{"issue":"10","key":"10.1007\/s13311-019-00810-8_bib75","doi-asserted-by":"crossref","first-page":"2061","DOI":"10.1038\/nprot.2013.126","article-title":"Seamless genome editing in human pluripotent stem cells using custom endonuclease-based gene targeting and the piggyBac transposon","volume":"8","author":"Yusa","year":"2013","journal-title":"Nat Protoc."},{"key":"10.1007\/s13311-019-00810-8_bib76","article-title":"Energy Metabolism and Mitochondrial Superoxide Anion Production in Pre-symptomatic Striatal Neurons Derived from Human-Induced Pluripotent Stem Cells Expressing Mutant Huntingtin","author":"Hamilton","year":"2019","journal-title":"Mol Neurobiol."},{"key":"10.1007\/s13311-019-00810-8_bib77","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1016\/j.lfs.2018.07.030","article-title":"Human cellular models of medium spiny neuron development and Huntington disease","volume":"209","author":"Golas","year":"2018","journal-title":"Life Sci."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib78","doi-asserted-by":"crossref","first-page":"e64368","DOI":"10.1371\/journal.pone.0064368","article-title":"Selective roles of normal and mutant huntingtin in neural induction and early neurogenesis","volume":"8","author":"Nguyen","year":"2013","journal-title":"PLoS One."},{"key":"10.1007\/s13311-019-00810-8_bib79","doi-asserted-by":"crossref","first-page":"76","DOI":"10.1016\/j.expneurol.2016.01.021","article-title":"Use of human stem cells in Huntington disease modeling and translational research","volume":"278","author":"Golas","year":"2016","journal-title":"Exp Neurol."},{"issue":"3","key":"10.1007\/s13311-019-00810-8_bib80","doi-asserted-by":"crossref","first-page":"275","DOI":"10.1038\/nbt.1529","article-title":"Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling","volume":"27","author":"Chambers","year":"2009","journal-title":"Nat Biotechnol."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib81","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1038\/nchembio.2007.54","article-title":"Dorsomorphin inhibits BMP signals required for embryogenesis and iron metabolism","volume":"4","author":"Yu","year":"2008","journal-title":"Nat Chem Biol."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib82","doi-asserted-by":"crossref","first-page":"e62721","DOI":"10.1371\/journal.pone.0062721","article-title":"A new class of small molecule inhibitor of BMP signaling","volume":"8","author":"Sanvitale","year":"2013","journal-title":"PLoS One."},{"issue":"15","key":"10.1007\/s13311-019-00810-8_bib83","doi-asserted-by":"crossref","first-page":"4388","DOI":"10.1016\/j.bmcl.2008.06.052","article-title":"Structure-activity relationship study of bone morphogenetic protein (BMP) signaling inhibitors","volume":"18","author":"Cuny","year":"2008","journal-title":"Bioorg Med Chem Lett."},{"issue":"12","key":"10.1007\/s13311-019-00810-8_bib84","doi-asserted-by":"crossref","first-page":"1129","DOI":"10.1038\/nbt1201-1129","article-title":"In vitro differentiation of transplantable neural precursors from human embryonic stem cells","volume":"19","author":"Zhang","year":"2001","journal-title":"Nat Biotechnol."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib85","doi-asserted-by":"crossref","first-page":"747","DOI":"10.1016\/0092-8674(95)90536-7","article-title":"Sonic hedgehog induces the differentiation of ventral forebrain neurons: a common signal for ventral patterning within the neural tube","volume":"81","author":"Ericson","year":"1995","journal-title":"Cell."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib86","doi-asserted-by":"crossref","first-page":"1097","DOI":"10.1242\/dev.013086","article-title":"Notochord-derived Shh concentrates in close association with the apically positioned basal body in neural target cells and forms a dynamic gradient during neural patterning","volume":"135","author":"Chamberlain","year":"2008","journal-title":"Development."},{"issue":"7","key":"10.1007\/s13311-019-00810-8_bib87","doi-asserted-by":"crossref","first-page":"683","DOI":"10.1038\/35083081","article-title":"Novel mechanism of Wnt signalling inhibition mediated by Dickkopf-1 interaction with LRP6\/Arrow","volume":"3","author":"Bafico","year":"2001","journal-title":"Nat Cell Biol."},{"issue":"6665","key":"10.1007\/s13311-019-00810-8_bib88","doi-asserted-by":"crossref","first-page":"357","DOI":"10.1038\/34848","article-title":"Dickkopf-1 is a member of a new family of secreted proteins and functions in head induction","volume":"391","author":"Glinka","year":"1998","journal-title":"Nature."},{"issue":"23","key":"10.1007\/s13311-019-00810-8_bib89","doi-asserted-by":"crossref","first-page":"4055","DOI":"10.1242\/dev.036624","article-title":"Coordination of sonic hedgehog and Wnt signaling determines ventral and dorsal telencephalic neuron types from human embryonic stem cells","volume":"136","author":"Li","year":"2009","journal-title":"Development."},{"issue":"7","key":"10.1007\/s13311-019-00810-8_bib90","doi-asserted-by":"crossref","first-page":"1375","DOI":"10.1242\/dev.117093","article-title":"Activin A directs striatal projection neuron differentiation of human pluripotent stem cells","volume":"142","author":"Arber","year":"2015","journal-title":"Development."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib91","doi-asserted-by":"crossref","first-page":"48","DOI":"10.1007\/s11689-009-9035-6","article-title":"Role for TGF-beta superfamily signaling in telencephalic GABAergic neuron development","volume":"2","author":"Maira","year":"2010","journal-title":"J Neurodev Disord."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib92","doi-asserted-by":"crossref","first-page":"455","DOI":"10.1016\/j.stem.2012.01.021","article-title":"Human embryonic stem cell-derived GABA neurons correct locomotion deficits in quinolinic acid-lesioned mice","volume":"10","author":"Ma","year":"2012","journal-title":"Cell Stem Cell."},{"issue":"9","key":"10.1007\/s13311-019-00810-8_bib93","doi-asserted-by":"crossref","first-page":"1763","DOI":"10.1002\/stem.1462","article-title":"Embryonic stem cells neural differentiation qualifies the role of Wnt\/beta-Catenin signals in human telencephalic specification and regionalization","volume":"31","author":"Nicoleau","year":"2013","journal-title":"Stem Cells."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib94","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1242\/dev.084608","article-title":"Developmentally coordinated extrinsic signals drive human pluripotent stem cell differentiation toward authentic DARPP-32+ medium-sized spiny neurons","volume":"140","author":"Delli Carri","year":"2013","journal-title":"Development."},{"key":"10.1007\/s13311-019-00810-8_bib95","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1186\/s13024-016-0092-5","article-title":"Manifestation of Huntington's disease pathology in human induced pluripotent stem cell-derived neurons","volume":"11","author":"Nekrasov","year":"2016","journal-title":"Mol Neurodegener."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib96","doi-asserted-by":"crossref","first-page":"1481","DOI":"10.1016\/j.stemcr.2018.03.007","article-title":"hPSC-Derived Striatal Cells Generated Using a Scalable 3D Hydrogel Promote Recovery in a Huntington Disease Mouse Model","volume":"10","author":"Adil","year":"2018","journal-title":"Stem Cell Reports."},{"issue":"22","key":"10.1007\/s13311-019-00810-8_bib97","doi-asserted-by":"crossref","first-page":"6583","DOI":"10.1113\/JP270655","article-title":"Improving and accelerating the differentiation and functional maturation of human stem cell-derived neurons: role of extracellular calcium and GABA","volume":"594","author":"Kemp","year":"2016","journal-title":"J Physiol."},{"issue":"5529","key":"10.1007\/s13311-019-00810-8_bib98","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1126\/science.1059581","article-title":"Loss of huntingtin-mediated BDNF gene transcription in Huntington's disease","volume":"293","author":"Zuccato","year":"2001","journal-title":"Science."},{"issue":"5-6","key":"10.1007\/s13311-019-00810-8_bib99","doi-asserted-by":"crossref","first-page":"294","DOI":"10.1016\/j.pneurobio.2007.01.003","article-title":"Role of brain-derived neurotrophic factor in Huntington's disease","volume":"81","author":"Zuccato","year":"2007","journal-title":"Prog Neurobiol."},{"issue":"13","key":"10.1007\/s13311-019-00810-8_bib100","doi-asserted-by":"crossref","first-page":"5409","DOI":"10.1523\/JNEUROSCI.19-13-05409.1999","article-title":"Expression of the striatal DARPP-32\/ARPP-21 phenotype in GABAergic neurons requires neurotrophins in vivo and in vitro","volume":"19","author":"Ivkovic","year":"1999","journal-title":"J Neurosci."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib101","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1007\/s004410050694","article-title":"Glial-cell-line-derived neurotrophic factor enhances biosynthesis of substance P in striatal neurons in vitro","volume":"286","author":"Humpel","year":"1996","journal-title":"Cell Tissue Res."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib102","doi-asserted-by":"crossref","first-page":"682","DOI":"10.1016\/j.ymthe.2004.02.016","article-title":"AAV-mediated gene delivery of BDNF or GDNF is neuroprotective in a model of Huntington disease","volume":"9","author":"Kells","year":"2004","journal-title":"Mol Ther."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib103","doi-asserted-by":"crossref","first-page":"461","DOI":"10.1007\/s12015-013-9441-8","article-title":"Human pluripotent stem cell differentiation into authentic striatal projection neurons","volume":"9","author":"Delli Carri","year":"2013","journal-title":"Stem Cell Rev."},{"issue":"7","key":"10.1007\/s13311-019-00810-8_bib104","doi-asserted-by":"crossref","first-page":"775","DOI":"10.5966\/sctm.2014-0083","article-title":"In Vitro Differentiation of Human Neural Progenitor Cells Into Striatal GABAergic Neurons","volume":"4","author":"Lin","year":"2015","journal-title":"Stem Cells Transl Med."},{"issue":"7","key":"10.1007\/s13311-019-00810-8_bib105","doi-asserted-by":"crossref","first-page":"E1234","DOI":"10.1073\/pnas.1611473114","article-title":"Differentiation of human telencephalic progenitor cells into MSNs by inducible expression of Gsx2 and Ebf1","volume":"114","author":"Faedo","year":"2017","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"3","key":"10.1007\/s13311-019-00810-8_bib106","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1038\/s41593-018-0075-7","article-title":"Striatal neurons directly converted from Huntington's disease patient fibroblasts recapitulate age-associated disease phenotypes","volume":"21","author":"Victor","year":"2018","journal-title":"Nat Neurosci."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib107","doi-asserted-by":"crossref","first-page":"311","DOI":"10.1016\/j.neuron.2014.10.016","article-title":"Generation of human striatal neurons by microRNA-dependent direct conversion of fibroblasts","volume":"84","author":"Victor","year":"2014","journal-title":"Neuron."},{"issue":"10","key":"10.1007\/s13311-019-00810-8_bib108","doi-asserted-by":"crossref","first-page":"1543","DOI":"10.1038\/nprot.2015.102","article-title":"MicroRNA-based conversion of human fibroblasts into striatal medium spiny neurons","volume":"10","author":"Richner","year":"2015","journal-title":"Nat Protoc."},{"issue":"3","key":"10.1007\/s13311-019-00810-8_bib109","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1016\/j.stemcr.2018.08.005","article-title":"A Chemical Recipe for Generation of Clinical-Grade Striatal Neurons from hESCs","volume":"11","author":"Wu","year":"2018","journal-title":"Stem Cell Reports."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib110","doi-asserted-by":"crossref","first-page":"703","DOI":"10.1016\/j.celrep.2012.04.009","article-title":"Generation of regionally specified neural progenitors and functional neurons from human embryonic stem cells under defined conditions","volume":"1","author":"Kirkeby","year":"2012","journal-title":"Cell Rep."},{"key":"10.1007\/s13311-019-00810-8_bib111","first-page":"64","article-title":"Generating regionalized neuronal cells from pluripotency, a step-by-step protocol","volume":"6","author":"Kirkeby","year":"2012","journal-title":"Front Cell Neurosci."},{"issue":"22","key":"10.1007\/s13311-019-00810-8_bib112","doi-asserted-by":"crossref","first-page":"14071","DOI":"10.1073\/pnas.182542899","article-title":"Small molecule modulation of Smoothened activity","volume":"99","author":"Chen","year":"2002","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib113","doi-asserted-by":"crossref","first-page":"1919","DOI":"10.1523\/JNEUROSCI.5128-10.2011","article-title":"Subregional specification of embryonic stem cell-derived ventral telencephalic tissues by timed and combinatory treatment with extrinsic signals","volume":"31","author":"Danjo","year":"2011","journal-title":"J Neurosci."},{"issue":"7264","key":"10.1007\/s13311-019-00810-8_bib114","doi-asserted-by":"crossref","first-page":"614","DOI":"10.1038\/nature08356","article-title":"Tankyrase inhibition stabilizes axin and antagonizes Wnt signalling","volume":"461","author":"Huang","year":"2009","journal-title":"Nature."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib115","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1016\/0022-510X(83)90053-9","article-title":"Nuclear membrane indentations in Huntington's chorea","volume":"61","author":"Roos","year":"1983","journal-title":"J Neurol Sci."},{"key":"10.1007\/s13311-019-00810-8_bib116","doi-asserted-by":"crossref","first-page":"696","DOI":"10.3389\/fphar.2018.00696","article-title":"Patient-Specific iPSC-Based Models of Huntington's Disease as a Tool to Study Store-Operated Calcium Entry Drug Targeting","volume":"9","author":"Vigont","year":"2018","journal-title":"Front Pharmacol."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib117","doi-asserted-by":"crossref","first-page":"80","DOI":"10.46582\/jsrm.1402012","article-title":"Mitochondrial distribution violation and nuclear indentations in neurons differentiated from iPSCs of Huntington's disease patients","volume":"14","author":"Nekrasov","year":"2018","journal-title":"J Stem Cells Regen Med."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib118","doi-asserted-by":"crossref","first-page":"191","DOI":"10.1016\/j.stemcr.2018.12.014","article-title":"Human Pluripotent Stem Cell-Derived Striatal Interneurons: Differentiation and Maturation In Vitro and in the Rat Brain","volume":"12","author":"Noakes","year":"2019","journal-title":"Stem Cell Reports."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib119","doi-asserted-by":"crossref","first-page":"1221","DOI":"10.1016\/j.stemcr.2017.08.019","article-title":"Scalable Production of iPSC-Derived Human Neurons to Identify Tau-Lowering Compounds by High-Content Screening","volume":"9","author":"Wang","year":"2017","journal-title":"Stem Cell Reports."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib120","doi-asserted-by":"crossref","first-page":"785","DOI":"10.1016\/j.neuron.2013.05.029","article-title":"Rapid single-step induction of functional neurons from human pluripotent stem cells","volume":"78","author":"Zhang","year":"2013","journal-title":"Neuron."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib121","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1186\/s40478-017-0475-z","article-title":"CRISPR\/Cas9-Correctable mutation-related molecular and physiological phenotypes in iPSC-derived Alzheimer's PSEN2 (N141I) neurons","volume":"5","author":"Ortiz-Virumbrales","year":"2017","journal-title":"Acta Neuropathol Commun."},{"issue":"31","key":"10.1007\/s13311-019-00810-8_bib122","doi-asserted-by":"crossref","first-page":"12770","DOI":"10.1073\/pnas.1202944109","article-title":"Modeling human cortical development in vitro using induced pluripotent stem cells","volume":"109","author":"Mariani","year":"2012","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib123","doi-asserted-by":"crossref","first-page":"1081","DOI":"10.1016\/j.celrep.2018.09.076","article-title":"Human Huntington's Disease iPSC-Derived Cortical Neurons Display Altered Transcriptomics, Morphology, and Maturation","volume":"25","author":"Mehta","year":"2018","journal-title":"Cell Rep."},{"issue":"10","key":"10.1007\/s13311-019-00810-8_bib124","doi-asserted-by":"crossref","first-page":"1836","DOI":"10.1038\/nprot.2012.116","article-title":"Directed differentiation of human pluripotent stem cells to cerebral cortex neurons and neural networks","volume":"7","author":"Shi","year":"2012","journal-title":"Nat Protoc."},{"issue":"12","key":"10.1007\/s13311-019-00810-8_bib125","doi-asserted-by":"crossref","first-page":"638","DOI":"10.1016\/j.tins.2009.08.002","article-title":"Molecular dissection of reactive astrogliosis and glial scar formation","volume":"32","author":"Sofroniew","year":"2009","journal-title":"Trends Neurosci."},{"key":"10.1007\/s13311-019-00810-8_bib126","doi-asserted-by":"crossref","first-page":"215","DOI":"10.3389\/fncel.2016.00215","article-title":"Astrocyte Differentiation of Human Pluripotent Stem Cells: New Tools for Neurological Disorder Research","volume":"10","author":"Chandrasekaran","year":"2016","journal-title":"Front Cell Neurosci."},{"key":"10.1007\/s13311-019-00810-8_bib127","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1186\/1756-6606-5-17","article-title":"Astrocytes generated from patient induced pluripotent stem cells recapitulate features of Huntington's disease patient cells","volume":"5","author":"Juopperi","year":"2012","journal-title":"Mol Brain."},{"issue":"8","key":"10.1007\/s13311-019-00810-8_bib128","doi-asserted-by":"crossref","first-page":"1325","DOI":"10.1097\/01.wnr.0000127073.66692.8f","article-title":"Autophagosome-like vacuole formation in Huntington's disease lymphoblasts","volume":"15","author":"Nagata","year":"2004","journal-title":"Neuroreport."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib129","doi-asserted-by":"crossref","first-page":"567","DOI":"10.1038\/nn.2528","article-title":"Cargo recognition failure is responsible for inefficient autophagy in Huntington's disease","volume":"13","author":"Martinez-Vicente","year":"2010","journal-title":"Nat Neurosci."},{"key":"10.1007\/s13311-019-00810-8_bib130","doi-asserted-by":"crossref","first-page":"669","DOI":"10.3389\/fnins.2019.00669","article-title":"Huntington's Disease Patient-Derived Astrocytes Display Electrophysiological Impairments and Reduced Neuronal Support","volume":"13","author":"Garcia","year":"2019","journal-title":"Front Neurosci."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib131","doi-asserted-by":"crossref","first-page":"160","DOI":"10.1002\/ana.24406","article-title":"Cerebrovascular and blood-brain barrier impairments in Huntington's disease: Potential implications for its pathophysiology","volume":"78","author":"Drouin-Ouellet","year":"2015","journal-title":"Ann Neurol."},{"issue":"7","key":"10.1007\/s13311-019-00810-8_bib132","doi-asserted-by":"crossref","first-page":"1365","DOI":"10.1016\/j.celrep.2017.04.021","article-title":"Huntington's Disease iPSC-Derived Brain Microvascular Endothelial Cells Reveal WNT-Mediated Angiogenic and Blood-Brain Barrier Deficits","volume":"19","author":"Lim","year":"2017","journal-title":"Cell Rep."},{"issue":"11","key":"10.1007\/s13311-019-00810-8_bib133","doi-asserted-by":"crossref","first-page":"1387","DOI":"10.1038\/nn1997","article-title":"Microglia: active sensor and versatile effector cells in the normal and pathologic brain","volume":"10","author":"Hanisch","year":"2007","journal-title":"Nat Neurosci."},{"issue":"10","key":"10.1007\/s13311-019-00810-8_bib134","doi-asserted-by":"crossref","first-page":"622","DOI":"10.1038\/s41583-018-0057-5","article-title":"Microglial signatures and their role in health and disease","volume":"19","author":"Butovsky","year":"2018","journal-title":"Nat Rev Neurosci."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib135","doi-asserted-by":"crossref","first-page":"513","DOI":"10.1038\/nn.3668","article-title":"Mutant Huntingtin promotes autonomous microglia activation via myeloid lineage-determining factors","volume":"17","author":"Crotti","year":"2014","journal-title":"Nat Neurosci."},{"issue":"16","key":"10.1007\/s13311-019-00810-8_bib136","doi-asserted-by":"crossref","first-page":"4328","DOI":"10.1093\/hmg\/ddu151","article-title":"Inhibition of soluble tumor necrosis factor is therapeutic in Huntington's disease","volume":"23","author":"Hsiao","year":"2014","journal-title":"Hum Mol Genet."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib137","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1038\/nn.3599","article-title":"Identification of a unique TGF-beta-dependent molecular and functional signature in microglia","volume":"17","author":"Butovsky","year":"2014","journal-title":"Nat Neurosci."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib138","doi-asserted-by":"crossref","first-page":"1727","DOI":"10.1016\/j.stemcr.2017.05.017","article-title":"A Highly Efficient Human Pluripotent Stem Cell Microglia Model Displays a Neuronal-Co-culture-Specific Expression Profile and Inflammatory Response","volume":"8","author":"Haenseler","year":"2017","journal-title":"Stem Cell Reports."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib139","doi-asserted-by":"crossref","first-page":"183","DOI":"10.1016\/j.immuni.2017.06.017","article-title":"Induced-Pluripotent-Stem-Cell-Derived Primitive Macrophages Provide a Platform for Modeling Tissue-Resident Macrophage Differentiation and Function","volume":"47","author":"Takata","year":"2017","journal-title":"Immunity."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib140","doi-asserted-by":"crossref","first-page":"753","DOI":"10.1038\/nn.4534","article-title":"Differentiation of human and murine induced pluripotent stem cells to microglia-like cells","volume":"20","author":"Pandya","year":"2017","journal-title":"Nat Neurosci."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib141","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1016\/j.neuron.2017.03.042","article-title":"iPSC-Derived Human Microglia-like Cells to Study Neurological Diseases","volume":"94","author":"Abud","year":"2017","journal-title":"Neuron."},{"issue":"11","key":"10.1007\/s13311-019-00810-8_bib142","doi-asserted-by":"crossref","first-page":"1358","DOI":"10.1038\/nm.4189","article-title":"Efficient derivation of microglia-like cells from human pluripotent stem cells","volume":"22","author":"Muffat","year":"2016","journal-title":"Nat Med."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib143","doi-asserted-by":"crossref","first-page":"1516","DOI":"10.1016\/j.stemcr.2017.04.023","article-title":"Directed Differentiation of Human Pluripotent Stem Cells to Microglia","volume":"8","author":"Douvaras","year":"2017","journal-title":"Stem Cell Reports."},{"key":"10.1007\/s13311-019-00810-8_bib144","doi-asserted-by":"crossref","DOI":"10.1002\/stem.2995","article-title":"Concise Review: Modeling Neurodegenerative Diseases with Human Pluripotent Stem Cell-Derived Microglia","author":"Haenseler","year":"2019","journal-title":"Stem Cells."},{"key":"10.1007\/s13311-019-00810-8_bib145","doi-asserted-by":"crossref","DOI":"10.1007\/s12035-019-1579-1","article-title":"Impaired Remyelination in a Mouse Model of Huntington Disease.","author":"Teo","year":"2019","journal-title":"Mol Neurobiol."},{"issue":"9","key":"10.1007\/s13311-019-00810-8_bib146","doi-asserted-by":"crossref","first-page":"2508","DOI":"10.1093\/hmg\/ddv016","article-title":"Early white matter abnormalities, progressive brain pathology and motor deficits in a novel knock-in mouse model of Huntington's disease","volume":"24","author":"Jin","year":"2015","journal-title":"Hum Mol Genet."},{"key":"10.1007\/s13311-019-00810-8_bib147","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1016\/j.brainres.2013.04.030","article-title":"Striatal oligodendrogliogenesis and neuroblast recruitment are increased in the R6\/2 mouse model of Huntington's disease","volume":"1518","author":"McCollum","year":"2013","journal-title":"Brain Res."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib148","doi-asserted-by":"crossref","first-page":"989","DOI":"10.1212\/01.WNL.0000138434.68093.67","article-title":"In vivo evidence of cerebellar atrophy and cerebral white matter loss in Huntington disease","volume":"63","author":"Fennema-Notestine","year":"2004","journal-title":"Neurology."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib149","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1097\/00005072-199111000-00005","article-title":"Decreased neuronal and increased oligodendroglial densities in Huntington's disease caudate nucleus","volume":"50","author":"Myers","year":"1991","journal-title":"J Neuropathol Exp Neurol."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib150","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1002\/1531-8249(200101)49:1<29::AID-ANA7>3.0.CO;2-B","article-title":"Quantitative neuropathological changes in presymptomatic Huntington's disease","volume":"49","author":"Gomez-Tortosa","year":"2001","journal-title":"Ann Neurol."},{"issue":"19","key":"10.1007\/s13311-019-00810-8_bib151","doi-asserted-by":"crossref","first-page":"9622","DOI":"10.1073\/pnas.1818042116","article-title":"Intrinsic mutant HTT-mediated defects in oligodendroglia cause myelination deficits and behavioral abnormalities in Huntington disease","volume":"116","author":"Ferrari Bardile","year":"2019","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib152","doi-asserted-by":"crossref","first-page":"1212","DOI":"10.1016\/j.neuron.2015.02.026","article-title":"Mutant huntingtin downregulates myelin regulatory factor-mediated myelin gene expression and affects mature oligodendrocytes","volume":"85","author":"Huang","year":"2015","journal-title":"Neuron."},{"issue":"11","key":"10.1007\/s13311-019-00810-8_bib153","doi-asserted-by":"crossref","first-page":"E2243","DOI":"10.1073\/pnas.1614412114","article-title":"Rapid and efficient generation of oligodendrocytes from human induced pluripotent stem cells using transcription factors","volume":"114","author":"Ehrlich","year":"2017","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib154","doi-asserted-by":"crossref","first-page":"250","DOI":"10.1016\/j.stemcr.2014.06.012","article-title":"Efficient generation of myelinating oligodendrocytes from primary progressive multiple sclerosis patients by induced pluripotent stem cells","volume":"3","author":"Douvaras","year":"2014","journal-title":"Stem Cell Reports."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib155","doi-asserted-by":"crossref","first-page":"519","DOI":"10.1016\/j.stem.2008.09.002","article-title":"Self-organized formation of polarized cortical tissues from ESCs and its active manipulation by extrinsic signals","volume":"3","author":"Eiraku","year":"2008","journal-title":"Cell Stem Cell."},{"issue":"7467","key":"10.1007\/s13311-019-00810-8_bib156","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1038\/nature12517","article-title":"Cerebral organoids model human brain development and microcephaly","volume":"501","author":"Lancaster","year":"2013","journal-title":"Nature."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib157","doi-asserted-by":"crossref","first-page":"375","DOI":"10.1016\/j.cell.2015.06.034","article-title":"FOXG1-Dependent Dysregulation of GABA\/Glutamate Neuron Differentiation in Autism Spectrum Disorders","volume":"162","author":"Mariani","year":"2015","journal-title":"Cell."},{"issue":"7","key":"10.1007\/s13311-019-00810-8_bib158","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1038\/nmeth.3415","article-title":"Functional cortical neurons and astrocytes from human pluripotent stem cells in 3D culture","volume":"12","author":"Pasca","year":"2015","journal-title":"Nat Methods."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib159","doi-asserted-by":"crossref","first-page":"E762","DOI":"10.1073\/pnas.1715865115","article-title":"Faulty neuronal determination and cell polarization are reverted by modulating HD early phenotypes","volume":"115","author":"Conforti","year":"2018","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib160","doi-asserted-by":"crossref","first-page":"e0175968","DOI":"10.1371\/journal.pone.0175968","article-title":"Peripheral huntingtin silencing does not ameliorate central signs of disease in the B6.HttQ111\/+ mouse model of Huntington's disease","volume":"12","author":"Coffey","year":"2017","journal-title":"PLoS One"},{"issue":"12","key":"10.1007\/s13311-019-00810-8_bib161","doi-asserted-by":"crossref","first-page":"3359","DOI":"10.1073\/pnas.1524575113","article-title":"Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis","volume":"113","author":"Wang","year":"2016","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib162","doi-asserted-by":"crossref","first-page":"1020","DOI":"10.1016\/j.celrep.2018.03.117","article-title":"Cardiac mTORC1 Dysregulation Impacts Stress Adaptation and Survival in Huntington's Disease","volume":"23","author":"Child","year":"2018","journal-title":"Cell Rep."},{"issue":"9","key":"10.1007\/s13311-019-00810-8_bib163","doi-asserted-by":"crossref","first-page":"2494","DOI":"10.1016\/j.celrep.2019.02.008","article-title":"Unbiased Profiling of Isogenic Huntington Disease hPSC-Derived CNS and Peripheral Cells Reveals Strong Cell-Type Specificity of CAG Length Effects","volume":"26","author":"Ooi","year":"2019","journal-title":"Cell Rep."},{"key":"10.1007\/s13311-019-00810-8_bib164","article-title":"Bioenergetic deficits in Huntington's disease iPSC-derived neural cells and rescue with glycolytic metabolites","author":"Kedaigle","year":"2019","journal-title":"Hum Mol Genet."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib165","doi-asserted-by":"crossref","first-page":"2886","DOI":"10.1038\/s41467-018-05320-3","article-title":"The ubiquitin ligase UBR5 suppresses proteostasis collapse in pluripotent stem cells from Huntington's disease patients","volume":"9","author":"Koyuncu","year":"2018","journal-title":"Nat Commun."},{"issue":"3","key":"10.1007\/s13311-019-00810-8_bib166","doi-asserted-by":"crossref","first-page":"359","DOI":"10.1042\/BJ20111495","article-title":"Quantitative proteomic analysis of induced pluripotent stem cells derived from a human Huntington's disease patient","volume":"446","author":"Chae","year":"2012","journal-title":"Biochem J."},{"issue":"7","key":"10.1007\/s13311-019-00810-8_bib167","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1016\/j.tibtech.2013.04.004","article-title":"ZFN, TALEN, and CRISPR\/Cas-based methods for genome engineering","volume":"31","author":"Gaj","year":"2013","journal-title":"Trends Biotechnol."},{"issue":"6213","key":"10.1007\/s13311-019-00810-8_bib168","doi-asserted-by":"crossref","first-page":"1258096","DOI":"10.1126\/science.1258096","article-title":"Genome editing. The new frontier of genome engineering with CRISPR-Cas9","volume":"346","author":"Doudna","year":"2014","journal-title":"Science"},{"key":"10.1007\/s13311-019-00810-8_bib169","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1007\/978-1-62703-761-7_16","article-title":"CRISPR-Cas-mediated targeted genome editing in human cells","volume":"1114","author":"Yang","year":"2014","journal-title":"Methods Mol Biol."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib170","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1016\/j.jgg.2013.12.004","article-title":"CRISPR\/Cas9 and genome editing in Drosophila","volume":"41","author":"Bassett","year":"2014","journal-title":"J Genet Genomics."},{"issue":"10","key":"10.1007\/s13311-019-00810-8_bib171","doi-asserted-by":"crossref","first-page":"1028","DOI":"10.1038\/nmeth.2641","article-title":"Engineering the Caenorhabditis elegans genome using Cas9-triggered homologous recombination","volume":"10","author":"Dickinson","year":"2013","journal-title":"Nat Methods."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib172","doi-asserted-by":"crossref","first-page":"653","DOI":"10.1016\/j.stem.2013.11.002","article-title":"Functional repair of CFTR by CRISPR\/Cas9 in intestinal stem cell organoids of cystic fibrosis patients","volume":"13","author":"Schwank","year":"2013","journal-title":"Cell Stem Cell."},{"issue":"6121","key":"10.1007\/s13311-019-00810-8_bib173","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1126\/science.1231143","article-title":"Multiplex genome engineering using CRISPR\/Cas systems","volume":"339","author":"Cong","year":"2013","journal-title":"Science."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib174","doi-asserted-by":"crossref","first-page":"399","DOI":"10.1038\/nmeth.2857","article-title":"Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects","volume":"11","author":"Shen","year":"2014","journal-title":"Nat Methods."},{"issue":"6","key":"10.1007\/s13311-019-00810-8_bib175","doi-asserted-by":"crossref","first-page":"1380","DOI":"10.1016\/j.cell.2013.08.021","article-title":"Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity","volume":"154","author":"Ran","year":"2013","journal-title":"Cell."},{"key":"10.1007\/s13311-019-00810-8_bib176","doi-asserted-by":"crossref","first-page":"75","DOI":"10.3389\/fnins.2018.00075","article-title":"Precise Excision of the CAG Tract from the Huntingtin Gene by Cas9 Nickases","volume":"12","author":"Dabrowska","year":"2018","journal-title":"Front Neurosci."},{"key":"10.1007\/s13311-019-00810-8_bib177","article-title":"Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR\/Cas9 System.","volume":"6","author":"An","year":"2014","journal-title":"PLoS Curr."},{"issue":"20","key":"10.1007\/s13311-019-00810-8_bib178","first-page":"4566","article-title":"Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR\/Cas9","volume":"25","author":"Shin","year":"2016","journal-title":"Hum Mol Genet."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib179","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1016\/j.ymthe.2016.11.010","article-title":"CRISPR\/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo","volume":"25","author":"Monteys","year":"2017","journal-title":"Mol Ther."},{"issue":"7","key":"10.1007\/s13311-019-00810-8_bib180","doi-asserted-by":"crossref","first-page":"2719","DOI":"10.1172\/JCI92087","article-title":"CRISPR\/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease","volume":"127","author":"Yang","year":"2017","journal-title":"J Clin Invest."},{"issue":"12","key":"10.1007\/s13311-019-00810-8_bib181","doi-asserted-by":"crossref","first-page":"8043","DOI":"10.1074\/jbc.M112.408211","article-title":"Enhanced aggregation of androgen receptor in induced pluripotent stem cell-derived neurons from spinal and bulbar muscular atrophy","volume":"288","author":"Nihei","year":"2013","journal-title":"J Biol Chem."},{"issue":"9","key":"10.1007\/s13311-019-00810-8_bib182","doi-asserted-by":"crossref","first-page":"1180","DOI":"10.1038\/nn.3787","article-title":"Polyglutamine-expanded androgen receptor interferes with TFEB to elicit autophagy defects in SBMA","volume":"17","author":"Cortes","year":"2014","journal-title":"Nat Neurosci."},{"key":"10.1007\/s13311-019-00810-8_bib183","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1016\/j.nbd.2014.05.038","article-title":"Stem cell-derived motor neurons from spinal and bulbar muscular atrophy patients","volume":"70","author":"Grunseich","year":"2014","journal-title":"Neurobiol Dis."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib184","doi-asserted-by":"crossref","first-page":"574","DOI":"10.1523\/JNEUROSCI.0906-12.2013","article-title":"Accelerated high-yield generation of limb-innervating motor neurons from human stem cells","volume":"33","author":"Amoroso","year":"2013","journal-title":"J Neurosci."},{"issue":"11","key":"10.1007\/s13311-019-00810-8_bib185","doi-asserted-by":"crossref","first-page":"1035","DOI":"10.1212\/WNL.0b013e31822e7ca0","article-title":"The natural history of spinocerebellar ataxia type 1, 2, 3, and 6: a 2-year follow-up study","volume":"77","author":"Jacobi","year":"2011","journal-title":"Neurology."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib186","doi-asserted-by":"crossref","first-page":"204","DOI":"10.1007\/s12311-008-0025-0","article-title":"Magnetic resonance imaging in spinocerebellar ataxias","volume":"7","author":"Dohlinger","year":"2008","journal-title":"Cerebellum."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib187","doi-asserted-by":"crossref","first-page":"1009","DOI":"10.1212\/WNL.49.4.1009","article-title":"The expansion of the CAG repeat in ataxin-2 is a frequent cause of autosomal dominant spinocerebellar ataxia","volume":"49","author":"Lorenzetti","year":"1997","journal-title":"Neurology."},{"issue":"3","key":"10.1007\/s13311-019-00810-8_bib188","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1038\/ng1196-277","article-title":"Identification of the spinocerebellar ataxia type 2 gene using a direct identification of repeat expansion and cloning technique","volume":"14","author":"Sanpei","year":"1996","journal-title":"DIRECT. Nat Genet."},{"issue":"3","key":"10.1007\/s13311-019-00810-8_bib189","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1038\/ng1196-285","article-title":"Cloning of the gene for spinocerebellar ataxia 2 reveals a locus with high sensitivity to expanded CAG\/glutamine repeats","volume":"14","author":"Imbert","year":"1996","journal-title":"Nat Genet."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib190","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1016\/j.jmb.2004.11.024","article-title":"An integrative approach to gain insights into the cellular function of human ataxin-2","volume":"346","author":"Ralser","year":"2005","journal-title":"J Mol Biol."},{"issue":"16","key":"10.1007\/s13311-019-00810-8_bib191","doi-asserted-by":"crossref","first-page":"2523","DOI":"10.1093\/hmg\/ddl173","article-title":"Ataxin-2 and its Drosophila homolog, ATX2, physically assemble with polyribosomes","volume":"15","author":"Satterfield","year":"2006","journal-title":"Hum Mol Genet."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib192","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1007\/s12031-012-9930-2","article-title":"Generation of human-induced pluripotent stem cells to model spinocerebellar ataxia type 2 in vitro","volume":"51","author":"Xia","year":"2013","journal-title":"J Mol Neurosci."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib193","doi-asserted-by":"crossref","first-page":"1166","DOI":"10.1038\/s41598-018-37774-2","article-title":"Modeling spinocerebellar ataxias 2 and 3 with iPSCs reveals a role for glutamate in disease pathology","volume":"9","author":"Chuang","year":"2019","journal-title":"Sci Rep."},{"key":"10.1007\/s13311-019-00810-8_bib194","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1007\/978-3-319-71779-1_11","article-title":"The Neuropathology of Spinocerebellar Ataxia Type 3\/Machado-Joseph Disease","volume":"1049","author":"Koeppen","year":"2018","journal-title":"Adv Exp Med Biol."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib195","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1055\/s-2007-971172","article-title":"Dominantly inherited ataxias: lessons learned from Machado-Joseph disease\/spinocerebellar ataxia type 3","volume":"27","author":"Paulson","year":"2007","journal-title":"Semin Neurol."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib196","doi-asserted-by":"crossref","first-page":"3605","DOI":"10.1074\/jbc.M310939200","article-title":"Poly-ubiquitin binding by the polyglutamine disease protein ataxin-3 links its normal function to protein surveillance pathways","volume":"279","author":"Chai","year":"2004","journal-title":"J Biol Chem."},{"issue":"7378","key":"10.1007\/s13311-019-00810-8_bib197","doi-asserted-by":"crossref","first-page":"543","DOI":"10.1038\/nature10671","article-title":"Excitation-induced ataxin-3 aggregation in neurons from patients with Machado-Joseph disease","volume":"480","author":"Koch","year":"2011","journal-title":"Nature."},{"key":"10.1007\/s13311-019-00810-8_bib198","doi-asserted-by":"crossref","first-page":"6701793","DOI":"10.1155\/2016\/6701793","article-title":"Autophagy Promoted the Degradation of Mutant ATXN3 in Neurally Differentiated Spinocerebellar Ataxia-3 Human Induced Pluripotent Stem Cells","volume":"2016","author":"Ou","year":"2016","journal-title":"Biomed Res Int."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib199","doi-asserted-by":"crossref","first-page":"306","DOI":"10.1016\/j.scr.2016.07.004","article-title":"Induced pluripotent stem cell - derived neurons for the study of spinocerebellar ataxia type 3","volume":"17","author":"Hansen","year":"2016","journal-title":"Stem Cell Res."},{"issue":"11","key":"10.1007\/s13311-019-00810-8_bib200","doi-asserted-by":"crossref","first-page":"756","DOI":"10.1089\/scd.2017.0209","article-title":"CRISPR\/Cas9-Targeted Deletion of Polyglutamine in Spinocerebellar Ataxia Type 3-Derived Induced Pluripotent Stem Cells","volume":"27","author":"Ouyang","year":"2018","journal-title":"Stem Cells Dev."},{"issue":"9","key":"10.1007\/s13311-019-00810-8_bib201","doi-asserted-by":"crossref","first-page":"3225","DOI":"10.1073\/pnas.0808387106","article-title":"A rosette-type, self-renewing human ES cell-derived neural stem cell with potential for in vitro instruction and synaptic integration","volume":"106","author":"Koch","year":"2009","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"11","key":"10.1007\/s13311-019-00810-8_bib202","doi-asserted-by":"crossref","first-page":"862","DOI":"10.5966\/sctm.2013-0080","article-title":"Efficient and rapid derivation of primitive neural stem cells and generation of brain subtype neurons from human pluripotent stem cells","volume":"2","author":"Yan","year":"2013","journal-title":"Stem Cells Transl Med."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib203","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1007\/s004010050787","article-title":"Neuropathological and molecular studies of spinocerebellar ataxia type 6 (SCA6)","volume":"95","author":"Sasaki","year":"1998","journal-title":"Acta Neuropathol."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib204","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/s00401-012-1000-x","article-title":"Brain pathology of spinocerebellar ataxias","volume":"124","author":"Seidel","year":"2012","journal-title":"Acta Neuropathol."},{"issue":"2","key":"10.1007\/s13311-019-00810-8_bib205","doi-asserted-by":"crossref","first-page":"336","DOI":"10.1086\/514867","article-title":"Japanese families with autosomal dominant pure cerebellar ataxia map to chromosome 19p13.1-p13.2 and are strongly associated with mild CAG expansions in the spinocerebellar ataxia type 6 gene in chromosome 19p13.1","volume":"61","author":"Ishikawa","year":"1997","journal-title":"Am J Hum Genet."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib206","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1016\/S0022-510X(98)00270-6","article-title":"Molecular analysis of a de novo mutation for spinocerebellar ataxia type 6 and (CAG)n repeat units in normal elder controls","volume":"161","author":"Shizuka","year":"1998","journal-title":"J Neurol Sci."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib207","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1038\/ng0197-62","article-title":"Autosomal dominant cerebellar ataxia (SCA6) associated with small polyglutamine expansions in the alpha 1A-voltage-dependent calcium channel","volume":"15","author":"Zhuchenko","year":"1997","journal-title":"Nat Genet."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib208","doi-asserted-by":"crossref","first-page":"118","DOI":"10.1016\/j.cell.2013.05.059","article-title":"Second cistron in CACNA1A gene encodes a transcription factor mediating cerebellar development and SCA6","volume":"154","author":"Du","year":"2013","journal-title":"Cell."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib209","doi-asserted-by":"crossref","first-page":"1075","DOI":"10.1016\/j.celrep.2017.01.012","article-title":"Vulnerability of Purkinje Cells Generated from Spinocerebellar Ataxia Type 6 Patient-Derived iPSCs","volume":"18","author":"Ishida","year":"2017","journal-title":"Cell Rep."},{"issue":"22","key":"10.1007\/s13311-019-00810-8_bib210","doi-asserted-by":"crossref","first-page":"1612","DOI":"10.1089\/scd.2017.0085","article-title":"Bicistronic CACNA1A Gene Expression in Neurons Derived from Spinocerebellar Ataxia Type 6 Patient-Induced Pluripotent Stem Cells","volume":"26","author":"Bavassano","year":"2017","journal-title":"Stem Cells Dev."},{"issue":"4","key":"10.1007\/s13311-019-00810-8_bib211","doi-asserted-by":"crossref","first-page":"537","DOI":"10.1016\/j.celrep.2014.12.051","article-title":"Self-organization of polarized cerebellar tissue in 3D culture of human pluripotent stem cells","volume":"10","author":"Muguruma","year":"2015","journal-title":"Cell Rep."},{"issue":"1","key":"10.1007\/s13311-019-00810-8_bib212","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1038\/ng0997-65","article-title":"Cloning of the SCA7 gene reveals a highly unstable CAG repeat expansion","volume":"17","author":"David","year":"1997","journal-title":"Nat Genet."},{"issue":"5","key":"10.1007\/s13311-019-00810-8_bib213","doi-asserted-by":"crossref","first-page":"1189","DOI":"10.1016\/j.celrep.2019.01.028","article-title":"Metabolic and Organelle Morphology Defects in Mice and Human Patients Define Spinocerebellar Ataxia Type 7 as a Mitochondrial Disease","volume":"26","author":"Ward","year":"2019","journal-title":"Cell Rep."},{"issue":"24","key":"10.1007\/s13311-019-00810-8_bib214","doi-asserted-by":"crossref","first-page":"2307","DOI":"10.1056\/NEJMoa1900907","article-title":"Targeting Huntingtin Expression in Patients with Huntington's Disease","volume":"380","author":"Tabrizi","year":"2019","journal-title":"N Engl J Med."},{"issue":"12","key":"10.1007\/s13311-019-00810-8_bib215","doi-asserted-by":"crossref","first-page":"1976","DOI":"10.1002\/mds.23176","article-title":"Impaired insulin sensitivity and secretion in normoglycemic patients with spinocerebellar ataxia type 1","volume":"25","author":"Lalic","year":"2010","journal-title":"Mov Disord."}],"container-title":["Neurotherapeutics"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s13311-019-00810-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/article\/10.1007\/s13311-019-00810-8\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1878747923009637?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1878747923009637?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s13311-019-00810-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,30]],"date-time":"2025-10-30T06:46:59Z","timestamp":1761806819000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1878747923009637"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,10]]},"references-count":215,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2019,10]]}},"alternative-id":["S1878747923009637"],"URL":"https:\/\/doi.org\/10.1007\/s13311-019-00810-8","relation":{},"ISSN":["1878-7479"],"issn-type":[{"value":"1878-7479","type":"print"}],"subject":[],"published":{"date-parts":[[2019,10]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Modeling Polyglutamine Expansion Diseases with Induced Pluripotent Stem Cells","name":"articletitle","label":"Article Title"},{"value":"Neurotherapeutics","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1007\/s13311-019-00810-8","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2019 The American Society for Experimental NeuroTherapeutics, Inc. Published by Elsevier B.V. All rights reserved.","name":"copyright","label":"Copyright"}]}}