{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,13]],"date-time":"2026-11-13T10:54:35Z","timestamp":1794567275363,"version":"4.9.1"},"publisher-location":"New York, NY","reference-count":116,"publisher":"Springer New York","isbn-type":[{"value":"9781493978243","type":"print"},{"value":"9781493978250","type":"electronic"}],"license":[{"start":{"date-parts":[[2018,1,1]],"date-time":"2018-01-01T00:00:00Z","timestamp":1514764800000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2018]]},"DOI":"10.1007\/978-1-4939-7825-0_23","type":"book-chapter","created":{"date-parts":[[2018,5,31]],"date-time":"2018-05-31T08:44:16Z","timestamp":1527756256000},"page":"497-523","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":37,"title":["Translating Antisense Technology into a Treatment for Huntington\u2019s Disease"],"prefix":"10.1007","author":[{"given":"Roger M.","family":"Lane","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne","family":"Smith","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tiffany","family":"Baumann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marc","family":"Gleichmann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dan","family":"Norris","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C. Frank","family":"Bennett","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Holly","family":"Kordasiewicz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2018,6,1]]},"reference":[{"key":"23_CR1","doi-asserted-by":"crossref","first-page":"215","DOI":"10.2174\/156720501103140329210642","volume":"11","author":"RE Becker","year":"2014","unstructured":"Becker RE, Greig NH (2014) A new regulatory road-map for Alzheimer\u2019s disease drug development. Curr Alzheimer Res 11:215\u2013220","journal-title":"Curr Alzheimer Res"},{"key":"23_CR2","doi-asserted-by":"crossref","first-page":"734","DOI":"10.1016\/S1474-4422(07)70178-3","volume":"6","author":"B Dubois","year":"2007","unstructured":"Dubois B, Feldman HH, Jacova C et al (2007) Research criteria for the diagnosis of Alzheimer\u2019s disease: revising the NINCDS-ADRDA criteria. Lancet Neurol 6:734\u2013746","journal-title":"Lancet Neurol"},{"key":"23_CR3","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1016\/j.parkreldis.2014.12.013","volume":"21","author":"D Zielonka","year":"2015","unstructured":"Zielonka D, Mielcarek M, Landwehrmeyer GB (2015) Update on Huntington\u2019s disease: advances in care and emerging therapeutic options. Parkinsonism Relat Disord 21:169\u2013178","journal-title":"Parkinsonism Relat Disord"},{"key":"23_CR4","doi-asserted-by":"crossref","first-page":"1434","DOI":"10.1002\/mds.26007","volume":"29","author":"EJ Wild","year":"2014","unstructured":"Wild EJ, Tabrizi SJ (2014) Targets for future clinical trials in Huntington\u2019s disease: what\u2019s in the pipeline? Mov Disord 29:1434\u20131445","journal-title":"Mov Disord"},{"issue":"16 Suppl","key":"23_CR5","volume":"86","author":"B Leavitt","year":"2016","unstructured":"Leavitt B, Kordasiewicz TS, Landwehrmeyer B et al (2016) Discovery and early clinical development of ISIS-HTTRx the first HTT-lowering drug to be tested in patients with Huntington\u2019s disease. Neurology 86(16 Suppl):PL01.002","journal-title":"Neurology"},{"key":"23_CR6","doi-asserted-by":"crossref","first-page":"1083","DOI":"10.1002\/mds.25075","volume":"27","author":"T Pringsheim","year":"2012","unstructured":"Pringsheim T, Wiltshire K, Day L et al (2012) The incidence and prevalence of Huntington\u2019s disease: a systematic review and meta-analysis. Mov Disord 27:1083\u20131091","journal-title":"Mov Disord"},{"key":"23_CR7","doi-asserted-by":"crossref","first-page":"11271133","DOI":"10.1002\/mds.23685","volume":"26","author":"I Shoulson","year":"2011","unstructured":"Shoulson I, Young AB (2011) Milestones in Huntington disease. Mov Disord 26:11271133","journal-title":"Mov Disord"},{"key":"23_CR8","doi-asserted-by":"crossref","first-page":"40","DOI":"10.1186\/1750-1172-5-40","volume":"5","author":"RA Roos","year":"2010","unstructured":"Roos RA (2010) Huntington\u2019s disease: a clinical review. Orphanet J Rare Dis 5:40","journal-title":"Orphanet J Rare Dis"},{"key":"23_CR9","doi-asserted-by":"crossref","first-page":"971","DOI":"10.1016\/0092-8674(93)90585-E","volume":"72","author":"The Huntington\u2019s Disease Collaborative Research Group","year":"1993","unstructured":"The Huntington\u2019s Disease Collaborative Research Group (1993) A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington\u2019s disease chromosomes. Cell 72:971\u2013983","journal-title":"Cell"},{"key":"23_CR10","doi-asserted-by":"crossref","first-page":"398","DOI":"10.1038\/ng0893-398","volume":"4","author":"SE Andrew","year":"1993","unstructured":"Andrew SE, Goldberg YP, Kremer B et al (1993) The relationship between trinucleotide (CAG) repeat length and clinical features of Huntington\u2019s disease. Nat Genet 4:398\u2013403","journal-title":"Nat Genet"},{"key":"23_CR11","doi-asserted-by":"crossref","first-page":"1714","DOI":"10.1002\/mds.25172","volume":"27","author":"F Squitieri","year":"2012","unstructured":"Squitieri F, Jankovic J (2012) Huntington\u2019s disease: how intermediate are intermediate repeat lengths? Mov Disord 27:1714\u20131717","journal-title":"Mov Disord"},{"key":"23_CR12","doi-asserted-by":"crossref","first-page":"2871","DOI":"10.1093\/hmg\/ddi319","volume":"14","author":"IS Seong","year":"2005","unstructured":"Seong IS, Ivanova E, Lee JM et al (2005) HD CAG repeat implicates a dominant property of huntingtin in mitochondrial energy metabolism. Hum Mol Genet 14:2871\u20132880","journal-title":"Hum Mol Genet"},{"key":"23_CR13","first-page":"217","volume":"26","author":"G Huntington","year":"1872","unstructured":"Huntington G (1872) On chorea. Med Surg Reporter 26:217\u2013321","journal-title":"Med Surg Reporter"},{"key":"23_CR14","doi-asserted-by":"crossref","first-page":"645","DOI":"10.1097\/00005053-198910000-00021","volume":"177","author":"S Folstein","year":"1989","unstructured":"Folstein S (1989) The psychopathology of Huntington\u2019s disease. J Nerv Ment Dis 177:645","journal-title":"J Nerv Ment Dis"},{"key":"23_CR15","doi-asserted-by":"crossref","first-page":"136","DOI":"10.1002\/mds.870110204","volume":"11","author":"The Huntington Study Group","year":"1996","unstructured":"The Huntington Study Group (1996) Unified Huntington\u2019s Disease Rating Scale: reliability and consistency. Mov Disord 11:136\u2013142","journal-title":"Mov Disord"},{"key":"23_CR16","doi-asserted-by":"crossref","first-page":"791","DOI":"10.1016\/S1474-4422(09)70170-X","volume":"8","author":"SJ Tabrizi","year":"2009","unstructured":"Tabrizi SJ, Langbehn DR, Leavitt BR et al (2009) Biological and clinical manifestations of Huntington\u2019s disease in the longitudinal TRACK-HD study: cross-sectional analysis of baseline data. Lancet Neurol 8:791\u2013801","journal-title":"Lancet Neurol"},{"key":"23_CR17","doi-asserted-by":"crossref","first-page":"204","DOI":"10.1038\/nrneurol.2014.24","volume":"10","author":"CA Ross","year":"2014","unstructured":"Ross CA, Aylward EH, Wild EJ et al (2014) Huntington disease: natural history biomarkers and prospects for therapeutics. Nat Rev Neurol 10:204\u2013216","journal-title":"Nat Rev Neurol"},{"key":"23_CR18","doi-asserted-by":"crossref","first-page":"961","DOI":"10.1111\/j.1460-9568.2006.04985.x","volume":"24","author":"A Petersen","year":"2006","unstructured":"Petersen A, Bjorkqvist M (2006) Hypothalamic-endocrine aspects in Huntington\u2019s disease. Eur J Neurosci 24:961\u2013967","journal-title":"Eur J Neurosci"},{"key":"23_CR19","doi-asserted-by":"crossref","first-page":"673","DOI":"10.1002\/mds.25864","volume":"29","author":"M Papoutsi","year":"2014","unstructured":"Papoutsi M, Labuschagne I, Tabrizi SJ, Stout JC (2014) The cognitive burden in Huntington\u2019s disease: pathology phenotype and mechanisms of compensation. Mov Disord 29:673\u2013683","journal-title":"Mov Disord"},{"key":"23_CR20","doi-asserted-by":"crossref","first-page":"1233","DOI":"10.1136\/jnnp-2013-305114","volume":"84","author":"JS Paulsen","year":"2013","unstructured":"Paulsen JS, Smith MM, Long JD (2013) Cognitive decline in prodromal Huntington Disease: implications for clinical trials. J Neurol Neurosurg Psychiatry 84:1233\u20131239","journal-title":"J Neurol Neurosurg Psychiatry"},{"key":"23_CR21","unstructured":"FDA Public Meeting on Patient-Focused Drug Development for Huntington\u2019s and Parkinson\u2019s Diseases (2015) September 22 (2015) January 22 (2017). Available from: http:\/\/www.fda.gov\/Drugs\/NewsEvents\/ucm451807.htm"},{"key":"23_CR22","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1002\/mds.20332","volume":"20","author":"P Hogarth","year":"2005","unstructured":"Hogarth P, Kayson E, Kieburtz K et al (2005) Interrater agreement in the assessment of motor manifestations of Huntington\u2019s disease. Mov Disord 20:293\u2013297","journal-title":"Mov Disord"},{"key":"23_CR23","doi-asserted-by":"crossref","first-page":"1335","DOI":"10.1002\/mds.26011","volume":"29","author":"R Reilmann","year":"2014","unstructured":"Reilmann R, Leavitt BR, Ross CA (2014) Diagnostic criteria for Huntington\u2019s disease based on natural history. Mov Disord 29:1335\u20131341","journal-title":"Mov Disord"},{"key":"23_CR24","doi-asserted-by":"crossref","first-page":"1417","DOI":"10.1126\/science.aad8709","volume":"352","author":"BA Sullenger","year":"2016","unstructured":"Sullenger BA, Nair S (2016) From the RNA world to the clinic. Science 352:1417\u20131420","journal-title":"Science"},{"key":"23_CR25","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1146\/annurev.pharmtox.010909.105654","volume":"50","author":"CF Bennett","year":"2010","unstructured":"Bennett CF, Swayze EE (2010) RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol 50:259\u2013293","journal-title":"Annu Rev Pharmacol Toxicol"},{"key":"23_CR26","doi-asserted-by":"crossref","first-page":"46","DOI":"10.1016\/j.addr.2015.01.008","volume":"87","author":"RS Geary","year":"2015","unstructured":"Geary RS, Norris D, Yu R, Bennett CF (2015) Pharmacokinetics biodistribution and cell uptake of antisense oligonucleotides. Adv Drug Deliv Rev 87:46\u201351","journal-title":"Adv Drug Deliv Rev"},{"key":"23_CR27","doi-asserted-by":"crossref","first-page":"35","DOI":"10.14304\/SURYA.JPR.V4N7.5","volume":"4","author":"J Chery","year":"2016","unstructured":"Chery J (2016) RNA therapeutics: RNAi and antisense mechanisms and clinical applications. Postdoc J 4:35\u201350","journal-title":"Postdoc J"},{"key":"23_CR28","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1124\/jpet.115.226969","volume":"355","author":"SK Pandey","year":"2015","unstructured":"Pandey SK, Wheeler TM, Justice SL et al (2015) Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1. J Pharmacol Exp Ther 355:329\u2013340","journal-title":"J Pharmacol Exp Ther"},{"key":"23_CR29","doi-asserted-by":"crossref","first-page":"46","DOI":"10.1124\/jpet.113.212407","volume":"350","author":"F Rigo","year":"2014","unstructured":"Rigo F, Chun SJ, Norris DA et al (2014) Pharmacology of a central nervous system delivered 2\u2032-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates. J Pharmacol Exp Ther 350:46\u201355","journal-title":"J Pharmacol Exp Ther"},{"key":"23_CR30","doi-asserted-by":"crossref","first-page":"941","DOI":"10.1016\/j.neuron.2016.04.042","volume":"90","author":"KM Schoch","year":"2016","unstructured":"Schoch KM, DeVos SL, Miller RL et al (2016) Increased 4r-tau induces pathological changes in a human-tau mouse model. Neuron 90:941\u2013947","journal-title":"Neuron"},{"key":"23_CR31","doi-asserted-by":"crossref","first-page":"13456","DOI":"10.1093\/nar\/gku1115","volume":"42","author":"WB Wan","year":"2014","unstructured":"Wan WB, Migawa MT, Vasquez G et al (2014) Synthesis biophysical properties and biological activity of second generation antisense oligonucleotides containing chiral phosphorothioate linkages. Nucleic Acids Res 42:13456\u201313468","journal-title":"Nucleic Acids Res"},{"key":"23_CR32","first-page":"327","volume-title":"Antisense drug technology","author":"ST Henry","year":"2008","unstructured":"Henry ST, Kim TW, Kramer-Stickland K et al (2008) Toxicologic properties of 2\u2032-o-methoxyethyl chimeric antisense inhibitors in animals and man. In: Crooke ST (ed) Antisense drug technology. Taylor & Francis Group, Boca Raton, pp 327\u2013364"},{"key":"23_CR33","doi-asserted-by":"crossref","first-page":"2239","DOI":"10.1016\/S0140-6736(16)31009-1","volume":"388","author":"NJ Viney","year":"2016","unstructured":"Viney NJ, van Capelleveen JC, Geary RS et al (2016) Antisense oligonucleotides targeting apolipoprotein(a) in people with raised lipoprotein(a): two randomised double-blind placebo-controlled dose-ranging trials. Lancet 388:2239\u20132253","journal-title":"Lancet"},{"key":"23_CR34","doi-asserted-by":"crossref","first-page":"8796","DOI":"10.1093\/nar\/gku531","volume":"42","author":"TP Prakash","year":"2014","unstructured":"Prakash TP, Graham MJ, Yu J et\u00a0al (2014) Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice. Nucleic Acids Res 42:8796\u20138807","journal-title":"Nucleic Acids Res"},{"key":"23_CR35","doi-asserted-by":"crossref","first-page":"899","DOI":"10.1177\/0883073815627882","volume":"31","author":"M Hache","year":"2016","unstructured":"Hache M, Swoboda KJ, Sethna N et al (2016) Intrathecal injections in children with spinal muscular atrophy: nusinersen clinical trial experience. J Child Neurol 31:899\u2013906","journal-title":"J Child Neurol"},{"key":"23_CR36","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1016\/S0076-6879(99)14099-0","volume":"314","author":"WC Broaddus","year":"2000","unstructured":"Broaddus WC, Prabhu SS, Wu-Pong S et al (2000) Strategies for the design and delivery of antisense oligonucleotides in central nervous system. Methods Enzymol 314:121\u2013135","journal-title":"Methods Enzymol"},{"key":"23_CR37","doi-asserted-by":"crossref","first-page":"1522","DOI":"10.1021\/mp300474m","volume":"10","author":"MI Papisov","year":"2013","unstructured":"Papisov MI, Belov VV, Gannon KS (2013) Physiology of the intrathecal bolus: the leptomeningeal route for macromolecule and particle delivery to CNS. Mol Pharm 10:1522\u20131532","journal-title":"Mol Pharm"},{"key":"23_CR38","doi-asserted-by":"crossref","first-page":"2583","DOI":"10.1007\/s11064-015-1581-6","volume":"40","author":"NA Jessen","year":"2015","unstructured":"Jessen NA, Munk AS, Lundgaard I, Nedergaard M (2015) The glymphatic system: a beginner\u2019s guide. Neurochem Res 40:2583\u20132599","journal-title":"Neurochem Res"},{"key":"23_CR39","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1016\/j.ymthe.2006.02.018","volume":"14","author":"P Hadaczek","year":"2006","unstructured":"Hadaczek P, Yamashita Y, Mirek H et al (2006) The \u201cperivascular pump\u201d driven by arterial pulsation is a powerful mechanism for the distribution of therapeutic molecules within the brain. Mol Ther 14:69\u201378","journal-title":"Mol Ther"},{"key":"23_CR40","doi-asserted-by":"crossref","first-page":"1031","DOI":"10.1016\/j.neuron.2012.05.009","volume":"74","author":"HB Kordasiewicz","year":"2012","unstructured":"Kordasiewicz HB, Stanek LM, Wancewicz EV et al (2012) Sustained therapeutic reversal of Huntington\u2019s disease by transient repression of huntingtin synthesis. Neuron 74:1031\u20131044","journal-title":"Neuron"},{"key":"23_CR41","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1016\/S1474-4422(13)70061-9","volume":"12","author":"TM Miller","year":"2013","unstructured":"Miller TM, Pestronk A, David W et al (2013) An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1 randomised first-in-man study. Lancet Neurol 12:435\u2013442","journal-title":"Lancet Neurol"},{"key":"23_CR42","doi-asserted-by":"crossref","first-page":"890","DOI":"10.1212\/WNL.0000000000002445","volume":"86","author":"CA Chiriboga","year":"2016","unstructured":"Chiriboga CA, Swoboda KJ, Darras BT et al (2016) Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy. Neurology 86:890\u2013897","journal-title":"Neurology"},{"key":"23_CR43","unstructured":"Spinraza (R) [Package Insert] Biogen Inc C MA; December (2016). Available from: wwwaccessdatafdagov\/drugsatfda_docs\/label\/(2016)\/209531lblpdf. Cited 15 Jan 2017"},{"key":"23_CR44","doi-asserted-by":"crossref","first-page":"217","DOI":"10.3233\/JHD-130057","volume":"2","author":"LM Stanek","year":"2013","unstructured":"Stanek LM, Yang W, Angus S et al (2013) Antisense oligonucleotide-mediated correction of transcriptional dysregulation is correlated with behavioral benefits in the YAC128 mouse model of Huntington\u2019s disease. J Huntingtons Dis 2:217\u2013228","journal-title":"J Huntingtons Dis"},{"key":"23_CR45","doi-asserted-by":"crossref","first-page":"842","DOI":"10.1002\/1531-8249(199912)46:6<842::AID-ANA6>3.0.CO;2-O","volume":"46","author":"S Kuemmerle","year":"1999","unstructured":"Kuemmerle S, Gutekunst CA, Klein AM et al (1999) Huntington aggregates may not predict neuronal death in Huntington\u2019s disease. Ann Neurol 46:842\u2013849","journal-title":"Ann Neurol"},{"key":"23_CR46","doi-asserted-by":"crossref","first-page":"127","DOI":"10.3389\/fneur.2013.00127","volume":"4","author":"L Moumne","year":"2013","unstructured":"Moumne L, Betuing S, Caboche J (2013) Multiple aspects of gene dysregulation in Huntington\u2019s disease. Front Neurol 4:127","journal-title":"Front Neurol"},{"key":"23_CR47","doi-asserted-by":"crossref","first-page":"1351","DOI":"10.1002\/mds.26012","volume":"29","author":"CA Ross","year":"2014","unstructured":"Ross CA, Pantelyat A, Kogan J, Brandt J (2014) Determinants of functional disability in Huntington\u2019s disease: Role of cognitive and motor dysfunction. Mov Disord 29:1351\u20131358","journal-title":"Mov Disord"},{"key":"23_CR48","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pgen.1002481","volume":"8","author":"M Banez-Coronel","year":"2012","unstructured":"Banez-Coronel M, Porta S, Kagerbauer B et al (2012) A pathogenic mechanism in Huntington\u2019s disease involves small CAG-repeated RNAs with neurotoxic activity. PLoS Genet 8:e1002481","journal-title":"PLoS Genet"},{"key":"23_CR49","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1038\/nrn1806","volume":"6","author":"E Cattaneo","year":"2005","unstructured":"Cattaneo E, Zuccato C, Tartari M (2005) Normal huntingtin function: an alternative approach to Huntington\u2019s disease. Nat Rev Neurosci 6:919\u2013930","journal-title":"Nat Rev Neurosci"},{"key":"23_CR50","doi-asserted-by":"crossref","first-page":"310","DOI":"10.1007\/s12311-012-0447-6","volume":"12","author":"H Tsoi","year":"2013","unstructured":"Tsoi H, Chan HY (2013) Expression of expanded CAG transcripts triggers nucleolar stress in Huntington\u2019s disease. Cerebellum 12:310\u2013312","journal-title":"Cerebellum"},{"key":"23_CR51","doi-asserted-by":"crossref","DOI":"10.1038\/ncomms11758","volume":"7","author":"A Benraiss","year":"2016","unstructured":"Benraiss A, Wang S, Herrlinger S et al (2016) Human glia can both induce and rescue aspects of disease phenotype in Huntington disease. Nat Commun 7:11758","journal-title":"Nat Commun"},{"issue":"5521","key":"23_CR52","doi-asserted-by":"crossref","first-page":"1552","DOI":"10.1126\/science.292.5521.1552","volume":"292","author":"NF Bence","year":"2001","unstructured":"Bence NF, Sampat RM, Kopito RR (2001) Impairment of the ubiquitin-proteasome system by protein aggregation. Science 292(5521):1552\u20131555","journal-title":"Science"},{"key":"23_CR53","doi-asserted-by":"crossref","first-page":"559","DOI":"10.1097\/00005072-198511000-00003","volume":"44","author":"JP Vonsattel","year":"1985","unstructured":"Vonsattel JP, Myers RH, Stevens TJ et al (1985) Neuropathological classification of Huntington\u2019s disease. J Neuropathol Exp Neurol 44:559\u2013577","journal-title":"J Neuropathol Exp Neurol"},{"key":"23_CR54","doi-asserted-by":"crossref","first-page":"19","DOI":"10.3389\/fncir.2013.00019","volume":"7","author":"AM Estrada-S\u00e1nchez","year":"2013","unstructured":"Estrada-S\u00e1nchez AM, Rebec GV (2013) Role of cerebral cortex in the neuropathology of Huntington\u2019s disease. Front Neural Circuits 7:19","journal-title":"Front Neural Circuits"},{"key":"23_CR55","doi-asserted-by":"crossref","first-page":"663","DOI":"10.1006\/exnr.1998.6919","volume":"154","author":"GM Halliday","year":"1998","unstructured":"Halliday GM, McRitchie DA, Macdonald V et al (1998) Regional specificity of brain atrophy in Huntington\u2019s disease. Exp Neurol 154:663\u2013672","journal-title":"Exp Neurol"},{"key":"23_CR56","doi-asserted-by":"crossref","first-page":"1094","DOI":"10.1093\/brain\/awq047","volume":"133","author":"DC Thu","year":"2010","unstructured":"Thu DC, Oorschot DE, Tippett LJ et al (2010) Cell loss in the motor and cingulate cortex correlates with symptomatology in Huntington\u2019s disease. Brain 133:1094\u20131110","journal-title":"Brain"},{"key":"23_CR57","doi-asserted-by":"crossref","first-page":"536","DOI":"10.1038\/nm.3514","volume":"20","author":"N Wang","year":"2014","unstructured":"Wang N, Gray M, Lu XH et al (2014) Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington\u2019s disease. Nat Med 20:536\u2013541","journal-title":"Nat Med"},{"key":"23_CR58","doi-asserted-by":"crossref","first-page":"8","DOI":"10.1186\/1750-1326-2-8","volume":"2","author":"X Gu","year":"2007","unstructured":"Gu X, Andr\u00e9 VM, Cepeda C et al (2007) Pathological cell-cell interactions are necessary for striatal pathogenesis in a conditional mouse model of Huntington\u2019s disease. Mol Neurodegener 2:8","journal-title":"Mol Neurodegener"},{"key":"23_CR59","doi-asserted-by":"crossref","first-page":"618","DOI":"10.1016\/j.ymthe.2005.05.006","volume":"12","author":"E Rodriguez-Lebron","year":"2005","unstructured":"Rodriguez-Lebron E, Denovan-Wright EM, Nash K et al (2005) Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6\/1 Huntington\u2019s disease transgenic mice. Mol Ther 12:618\u2013633","journal-title":"Mol Ther"},{"key":"23_CR60","doi-asserted-by":"crossref","first-page":"1025","DOI":"10.1093\/hmg\/ddl017","volume":"15","author":"H Wang","year":"2006","unstructured":"Wang H, Lim PJ, Yin C et al (2006) Suppression of polyglutamine-induced toxicity in cell and animal models of Huntington\u2019s disease by ubiquitin. Hum Mol Genet 15:1025\u20131041","journal-title":"Hum Mol Genet"},{"key":"23_CR61","doi-asserted-by":"crossref","first-page":"1053","DOI":"10.1038\/mt.2009.17","volume":"17","author":"RL Boudreau","year":"2009","unstructured":"Boudreau RL, McBride JL, Martins I et al (2009) Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington\u2019s disease mice. Mol Ther 17:1053\u20131063","journal-title":"Mol Ther"},{"key":"23_CR62","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1016\/S0092-8674(00)80623-6","volume":"101","author":"A Yamamoto","year":"2000","unstructured":"Yamamoto A, Lucas JJ, Hen R (2000) Reversal of neuropathology and motor dysfunction in a conditional model of Huntington\u2019s disease. Cell 101:57\u201366","journal-title":"Cell"},{"key":"23_CR63","doi-asserted-by":"crossref","first-page":"5820","DOI":"10.1073\/pnas.0501507102","volume":"102","author":"SQ Harper","year":"2005","unstructured":"Harper SQ, Staber PD, He X et al (2005) RNA interference improves motor and neuropathological abnormalities in a Huntington\u2019s disease mouse model. Proc Natl Acad Sci U S A 102:5820\u20135825","journal-title":"Proc Natl Acad Sci U S A"},{"key":"23_CR64","doi-asserted-by":"crossref","first-page":"9773","DOI":"10.1523\/JNEUROSCI.3183-05.2005","volume":"25","author":"M Diaz-Hernandez","year":"2005","unstructured":"Diaz-Hernandez M, Torres-Peraza J, Salvatori-Abarca A et al (2005) Full motor recovery despite striatal neuron loss and formation of irreversible amyloid-like inclusions in a conditional mouse model of Huntington\u2019s disease. J Neurosci 25:9773\u20139781","journal-title":"J Neurosci"},{"key":"23_CR65","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1038\/nbt.1539","volume":"27","author":"J Hu","year":"2009","unstructured":"Hu J, Matsui M, Gagnon KT et al (2009) Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs. Nat Biotechnol 27:478\u2013484","journal-title":"Nat Biotechnol"},{"key":"23_CR66","doi-asserted-by":"crossref","first-page":"774","DOI":"10.1016\/j.cub.2009.03.030","volume":"19","author":"EL Pfister","year":"2009","unstructured":"Pfister EL, Kennington L, Straubhaar J et\u00a0al (2009) Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington\u2019s disease patients. Curr Biol 19:774\u2013778","journal-title":"Curr Biol"},{"key":"23_CR67","doi-asserted-by":"crossref","first-page":"1183","DOI":"10.1016\/j.chembiol.2010.10.013","volume":"17","author":"J Hu","year":"2010","unstructured":"Hu J, Liu J, Corey DR (2010) Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism. Chem Biol 17:1183\u20131188","journal-title":"Chem Biol"},{"key":"23_CR68","doi-asserted-by":"crossref","first-page":"2093","DOI":"10.1038\/mt.2014.153","volume":"22","author":"AL Southwell","year":"2014","unstructured":"Southwell AL, Skotte NH, Kordasiewicz HB et al (2014) In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides. Mol Ther 22:2093\u20132106","journal-title":"Mol Ther"},{"key":"23_CR69","doi-asserted-by":"crossref","first-page":"5736","DOI":"10.1073\/pnas.1603871113","volume":"113","author":"AE Molero","year":"2016","unstructured":"Molero AE, Arteaga-Bracho EE, Chen CH et al (2016) Selective expression of mutant huntingtin during development recapitulates characteristic features of Huntington\u2019s disease. Proc Natl Acad Sci U S A 113:5736\u20135741","journal-title":"Proc Natl Acad Sci U S A"},{"key":"23_CR70","doi-asserted-by":"crossref","first-page":"2366","DOI":"10.1073\/pnas.1221891110","volume":"110","author":"K Sathasivam","year":"2013","unstructured":"Sathasivam K, Neueder A, Gipson TA et al (2013) Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington disease. Proc Natl Acad Sci U S A 110:2366\u20132370","journal-title":"Proc Natl Acad Sci U S A"},{"key":"23_CR71","doi-asserted-by":"crossref","first-page":"913","DOI":"10.1093\/hmg\/ddu504","volume":"24","author":"BA Barbaro","year":"2014","unstructured":"Barbaro BA, Lukacsovich T, Agrawal N et al (2014) Comparative study of naturally occurring huntingtin fragments in Drosophila points to exon 1 as the most pathogenic species in Huntington\u2019s disease. Hum Mol Genet 24:913\u2013925","journal-title":"Hum Mol Genet"},{"key":"23_CR72","doi-asserted-by":"crossref","first-page":"215","DOI":"10.1089\/oli.1.1998.8.215","volume":"8","author":"AJ Cheng","year":"1998","unstructured":"Cheng AJ, Wang JC, Van Dyke MW (1998) Self-association of G-rich oligodeoxyribonucleotides under conditions promoting purine-motif triplex formation. Antisense Nucleic Acid Drug Dev 8:215\u2013225","journal-title":"Antisense Nucleic Acid Drug Dev"},{"key":"23_CR73","doi-asserted-by":"crossref","first-page":"709","DOI":"10.1146\/annurev.immunol.20.100301.064842","volume":"20","author":"AM Krieg","year":"2002","unstructured":"Krieg AM (2002) CpG motifs in bacterial DNA and their immune effects. Annu Rev Immunol 20:709\u2013760","journal-title":"Annu Rev Immunol"},{"key":"23_CR74","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1007\/BF02290678","volume":"20","author":"G Barnes","year":"1994","unstructured":"Barnes G, Duyao MP, Ambrose CM et al (1994) Mouse Huntington\u2019s disease gene homolog. Somat Cell Mol Genet 20:87\u201397","journal-title":"Somat Cell Mol Genet"},{"key":"23_CR75","first-page":"2179","volume":"9","author":"R Chang","year":"2015","unstructured":"Chang R, Liu X, Li S, Li XJ (2015) Transgenic animal models for study of the pathogenesis of Huntington\u2019s disease and therapy. Drug Des Devel Ther 9:2179\u20132188","journal-title":"Drug Des Devel Ther"},{"key":"23_CR76","doi-asserted-by":"crossref","first-page":"2178","DOI":"10.1038\/mt.2011.201","volume":"19","author":"JB Carroll","year":"2011","unstructured":"Carroll JB, Warby SC, Southwell AL et al (2011) Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene\/allele-specific silencing of mutant huntingtin. Mol Ther 19:2178\u20132185","journal-title":"Mol Ther"},{"key":"23_CR77","doi-asserted-by":"crossref","first-page":"6182","DOI":"10.1523\/JNEUROSCI.0857-08.2008","volume":"28","author":"M Gray","year":"2008","unstructured":"Gray M, Shirasaki DI, Cepeda C et al (2008) Full-length human mutant huntingtin with a stable polyglutamine repeat can elicit progressive and selective neuropathogenesis in BACHD mice. J Neurosci 28:6182\u20136195","journal-title":"J Neurosci"},{"key":"23_CR78","doi-asserted-by":"crossref","first-page":"303","DOI":"10.1007\/978-1-4939-1221-6_9","volume":"825","author":"F Rigo","year":"2014","unstructured":"Rigo F, Seth PP, Bennett CF (2014) Antisense oligonucleotide-based therapies for diseases caused by pre-mRNA processing defects. Adv Exp Med Biol 825:303\u2013352","journal-title":"Adv Exp Med Biol"},{"key":"23_CR79","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1080\/17460441.2017.1271320","volume":"12","author":"D Dam Van","year":"2017","unstructured":"Van Dam D, De Deyn PP (2017) Non human primate models for Alzheimer\u2019s disease-related research and drug discovery. Expert Opin Drug Discov 12:187\u2013200","journal-title":"Expert Opin Drug Discov"},{"key":"23_CR80","doi-asserted-by":"crossref","first-page":"115","DOI":"10.1007\/978-3-662-46943-9_5","volume":"229","author":"MJ Kallman","year":"2015","unstructured":"Kallman MJ (2015) Preclinical abuse potential assessment. Handb Exp Pharmacol 229:115\u2013130","journal-title":"Handb Exp Pharmacol"},{"key":"23_CR81","first-page":"1444","volume":"2","author":"SP Henry","year":"2001","unstructured":"Henry SP, Geary RS, Yu R et al (2001) Drug properties of second-generation antisense oligonucleotides: how do they measure up to their predecessors? Curr Opin Investig Drugs 2:1444\u20131449","journal-title":"Curr Opin Investig Drugs"},{"key":"23_CR82","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1089\/nat.2015.0588","volume":"26","author":"R Yu","year":"2016","unstructured":"Yu R, Warren MS, Watanabe TZ et al (2016) Lack of interactions between an antisense oligonucleotide with 2\u2032-O-(2-methoxyethyl) modifications and major drug transporters. Nucleic Acid Ther 26:111\u2013117","journal-title":"Nucleic Acid Ther"},{"key":"23_CR83","doi-asserted-by":"crossref","first-page":"972","DOI":"10.1124\/jpet.105.084004","volume":"314","author":"JJ Senn","year":"2005","unstructured":"Senn JJ, Burel S, Henry SP (2005) Non-CpG-containing antisense 2\u2032-methoxyethyl oligonucleotides activate a proinflammatory response independent of Toll-like receptor 9 or myeloid differentiation factor 88. J Pharmacol Exp Ther 314:972\u2013979","journal-title":"J Pharmacol Exp Ther"},{"key":"23_CR84","doi-asserted-by":"crossref","first-page":"326","DOI":"10.1089\/nat.2014.0491","volume":"24","author":"SP Henry","year":"2014","unstructured":"Henry SP, Jagels MA, Hugli TE et al (2014) Mechanism of alternative complement pathway dysregulation by a phosphorothioate oligonucleotide in monkey and human serum. Nucleic Acid Ther 24:326\u2013335","journal-title":"Nucleic Acid Ther"},{"key":"23_CR85","doi-asserted-by":"crossref","first-page":"1","DOI":"10.3233\/JHD-160196","volume":"5","author":"LM Byrne","year":"2016","unstructured":"Byrne LM, Wild EJ (2016) Cerebrospinal fluid biomarkers for Huntington\u2019s disease. J Huntingtons Dis 5:1\u201313","journal-title":"J Huntingtons Dis"},{"key":"23_CR86","doi-asserted-by":"crossref","first-page":"1419","DOI":"10.1124\/dmd.31.11.1419","volume":"31","author":"RS Geary","year":"2003","unstructured":"Geary RS, Yu RZ, Watanabe T et al (2003) Pharmacokinetics of a tumor necrosis factor-alpha phosphorothioate 2\u2032-O-(2-methoxyethyl) modified antisense oligonucleotide: comparison across species. Drug Metab Dispos 31:1419\u20131428","journal-title":"Drug Metab Dispos"},{"key":"23_CR87","doi-asserted-by":"crossref","first-page":"460","DOI":"10.1124\/dmd.106.012401","volume":"35","author":"RZ Yu","year":"2007","unstructured":"Yu RZ, Kim TW, Hong A et al (2007) Cross-species pharmacokinetic comparison from mouse to man of a second-generation antisense oligonucleotide ISIS 301012 targeting human apolipoprotein B-100. Drug Metab Dispos 35:460\u2013468","journal-title":"Drug Metab Dispos"},{"key":"23_CR88","volume":"4","author":"RZ Yu","year":"2015","unstructured":"Yu RZ, Grundy JS, Henry SP et al (2015) Predictive dose-based estimation of systemic exposure multiples in mouse and monkey relative to human for antisense oligonucleotides with 2\u2032-o-(2-methoxyethyl) modifications. Mol Ther Nucleic Acids 4:e218","journal-title":"Mol Ther Nucleic Acids"},{"key":"23_CR89","unstructured":"European Medicines Agency (2016) Guideline on strategies to identify and mitigate risks for first-in-human and early clinical trials with investigational medicinal products in EMEA\/CHMP\/SWP\/28367\/07 Rev 1 EMACfMPfHU (CHMP) Editor"},{"key":"23_CR90","doi-asserted-by":"crossref","first-page":"1364","DOI":"10.2522\/ptj.20150580","volume":"96","author":"FB Horak","year":"2016","unstructured":"Horak FB, Mancini M, Carlson-Kuhta P et al (2016) Balance and gait represent independent domains of mobility in Parkinson disease. Phys Ther 96:1364\u20131371","journal-title":"Phys Ther"},{"key":"23_CR91","doi-asserted-by":"crossref","first-page":"1019","DOI":"10.1139\/apnm-2015-0140","volume":"40","author":"R Villar","year":"2015","unstructured":"Villar R, Beltrame T, Hughson RL (2015) Validation of the Hexoskin wearable vest during lying sitting standing and walking activities. Appl Physiol Nutr Metab 40:1019\u20131024","journal-title":"Appl Physiol Nutr Metab"},{"issue":"3\u20134","key":"23_CR92","first-page":"79","volume":"21","author":"H Reiber","year":"2003","unstructured":"Reiber H (2003) Proteins in cerebrospinal fluid and blood: barriers CSF flow rate and source-related dynamics. Restor Neurol Neurosci 21(3\u20134):79\u201396","journal-title":"Restor Neurol Neurosci"},{"key":"23_CR93","unstructured":"Wild EJ, Boborwsky B, HDClarity investigators (2016) A new multi-site cerebrospinal fluid collection initiative to facilitate therapeutic development for Huntington disease. In: 11th annual CHDI HD therapeutics conference, Palm Springs, CA"},{"key":"23_CR94","doi-asserted-by":"crossref","first-page":"1970","DOI":"10.1002\/elps.200600752","volume":"28","author":"A Schrattenholz","year":"2007","unstructured":"Schrattenholz A, Groebe K (2007) What does it need to be a biomarker? Relationships between resolution differential quantification and statistical validation of protein surrogate biomarkers. Electrophoresis 28:1970\u20131979","journal-title":"Electrophoresis"},{"key":"23_CR95","doi-asserted-by":"crossref","first-page":"1979","DOI":"10.1172\/JCI80743","volume":"125","author":"EJ Wild","year":"2015","unstructured":"Wild EJ, Boggio R, Langbehn D et al (2015) Quantification of mutant huntingtin protein in cerebrospinal fluid from Huntington\u2019s disease patients. J Clin Invest 125:1979\u20131986","journal-title":"J Clin Invest"},{"key":"23_CR96","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1016\/j.parkreldis.2008.05.012","volume":"15","author":"R Constantinescu","year":"2009","unstructured":"Constantinescu R, Romer M, Zetterberg H et al (2009) Levels of the light subunit of neurofilament triplet protein in cerebrospinal fluid in Huntington\u2019s disease. Parkinsonism Relat Disord 15:245\u2013248","journal-title":"Parkinsonism Relat Disord"},{"key":"23_CR97","doi-asserted-by":"crossref","first-page":"714","DOI":"10.1016\/j.parkreldis.2011.06.010","volume":"17","author":"R Constantinescu","year":"2011","unstructured":"Constantinescu R, Romer M, Zetterberg H et al (2011) Increased levels of total tau protein in the cerebrospinal fluid in Huntington\u2019s disease. Parkinsonism Relat Disord 17:714\u2013715","journal-title":"Parkinsonism Relat Disord"},{"key":"23_CR98","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1111\/jnc.13719","volume":"139","author":"FB Rodrigues","year":"2016","unstructured":"Rodrigues FB, Byrne L, McColgan P et al (2016) Cerebrospinal fluid total tau concentration predicts clinical phenotype in Huntington\u2019s disease. J Neurochem 139:22\u201325","journal-title":"J Neurochem"},{"key":"23_CR99","doi-asserted-by":"crossref","first-page":"1184","DOI":"10.15252\/emmm.201606540","volume":"8","author":"N Mattsson","year":"2016","unstructured":"Mattsson N, Insel PS, Palmqvist S et al (2016) Cerebrospinal fluid tau neurogranin and neurofilament light in Alzheimer\u2019s disease. EMBO Mol Med 8:1184\u20131196","journal-title":"EMBO Mol Med"},{"key":"23_CR100","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1016\/j.conb.2015.01.020","volume":"33","author":"JL Plotkin","year":"2015","unstructured":"Plotkin JL, Surmeier DJ (2015) Corticostriatal synaptic adaptations in Huntington\u2019s disease. Curr Opin Neurobiol 33:53\u201362","journal-title":"Curr Opin Neurobiol"},{"key":"23_CR101","volume":"11","author":"FB Rodrigues","year":"2016","unstructured":"Rodrigues FB, Byrne LM, McColgan P et al (2016) Cerebrospinal fluid inflammatory biomarkers reflect clinical severity in Huntington\u2019s disease. PLoS One 11:e0163479","journal-title":"PLoS One"},{"key":"23_CR102","doi-asserted-by":"crossref","first-page":"78","DOI":"10.3389\/fnagi.2014.00078","volume":"6","author":"JS Paulsen","year":"2014","unstructured":"Paulsen JS, Long JD, Johnson HJ et al (2014) Clinical and biomarker changes in premanifest Huntington disease show trial feasibility: a decade of the PREDICT-HD study. Front Aging Neurosci 6:78","journal-title":"Front Aging Neurosci"},{"key":"23_CR103","doi-asserted-by":"crossref","first-page":"1411","DOI":"10.1136\/jnnp-2013-307343","volume":"85","author":"E Duijn van","year":"2014","unstructured":"van Duijn E, Craufurd D, Hubers AA et al (2014) Neuropsychiatric symptoms in a European Huntington\u2019s disease cohort (REGISTRY). J Neurol Neurosurg Psychiatry 85:1411\u20131418","journal-title":"J Neurol Neurosurg Psychiatry"},{"key":"23_CR104","doi-asserted-by":"crossref","first-page":"204","DOI":"10.1016\/j.nicl.2012.12.001","volume":"2","author":"NZ Hobbs","year":"2012","unstructured":"Hobbs NZ, Henley NZ, Ridgway GR et al (2012) Evaluation of multi-modal multi-site neuroimaging measures in Huntington\u2019s disease: baseline results from the PADDINGTON study. Neuroimage Clin 2:204\u2013211","journal-title":"Neuroimage Clin"},{"key":"23_CR105","doi-asserted-by":"crossref","first-page":"42","DOI":"10.1016\/S1474-4422(11)70263-0","volume":"11","author":"SJ Tabrizi","year":"2012","unstructured":"Tabrizi SJ, Reilmann R, Roos RA et al (2012) Potential endpoints for clinical trials in premanifest and early Huntington\u2019s disease in the TRACK-HD study: analysis of 24 month observational data. Lancet Neurol 11:42\u201353","journal-title":"Lancet Neurol"},{"key":"23_CR106","doi-asserted-by":"crossref","first-page":"756","DOI":"10.1136\/jnnp.2009.190702","volume":"81","author":"NZ Hobbs","year":"2010","unstructured":"Hobbs NZ, Henley SM, Ridgeway GE et al (2010) The progression of regional atrophy in premanifest and early Huntington\u2019s disease: a longitudinal voxel-based morphometry study. J Neurol Neurosurg Psychiatry 81:756\u2013763","journal-title":"J Neurol Neurosurg Psychiatry"},{"key":"23_CR107","doi-asserted-by":"crossref","first-page":"548","DOI":"10.1016\/j.tins.2009.06.003","volume":"32","author":"D Eidelberg","year":"2009","unstructured":"Eidelberg D (2009) Metabolic brain networks in neurodegenerative disorders: a functional imaging approach. Trends Neurosci 32:548\u2013557","journal-title":"Trends Neurosci"},{"key":"23_CR108","doi-asserted-by":"crossref","first-page":"1420","DOI":"10.1016\/j.ebiom.2015.08.002","volume":"2","author":"S Kl\u00f6ppel","year":"2015","unstructured":"Kl\u00f6ppel S, Gregory S, Scheller E et al (2015) Compensation in preclinical Huntington\u2019s disease: evidence from the Track-on HD study. EBioMedicine 2:1420\u20131429","journal-title":"EBioMedicine"},{"key":"23_CR109","unstructured":"Sasinowski FJ (2011) Quantum of effectiveness evidence in FDA\u2019s approval of orphan drugs: cataloguing FDA\u2019s flexibility in regulating therapies for persons with rare disorders. National Organization for Rare Disorders:1\u201327"},{"key":"23_CR110","unstructured":"FDA (2014) CFR-21 340510: Accelerated approval of new drugs for serious or life-threatening illnesses"},{"key":"23_CR111","doi-asserted-by":"crossref","first-page":"1281","DOI":"10.1002\/mds.25964","volume":"29","author":"JC Stout","year":"2014","unstructured":"Stout JC, Queller S, Baker KN et al (2014) HD-CAB: a cognitive assessment battery for clinical trials in Huntington\u2019s disease 123. Mov Disord 29:1281\u20131288","journal-title":"Mov Disord"},{"key":"23_CR112","doi-asserted-by":"crossref","first-page":"1419","DOI":"10.1002\/mds.26021","volume":"29","author":"C Sampaio","year":"2014","unstructured":"Sampaio C, Borowsky B, Reilmann R (2014) Clinical trials in Huntington\u2019s disease: interventions in early clinical development and newer methodological approaches. Mov Disord 29:1419\u20131428","journal-title":"Mov Disord"},{"key":"23_CR113","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pbio.1002525","volume":"14","author":"TJ Little","year":"2016","unstructured":"Little TJ, Colegrave N (2016) Caging and uncaging genetics. PLoS Biol 14:e1002525","journal-title":"PLoS Biol"},{"key":"23_CR114","doi-asserted-by":"crossref","first-page":"1253","DOI":"10.1016\/j.neuron.2016.08.013","volume":"91","author":"LJ Sittig","year":"2016","unstructured":"Sittig LJ, Carbonetto P, Engel KA et al (2016) Genetic background limits generalizability of genotype-phenotype relationships. Neuron 91:1253\u20131259","journal-title":"Neuron"},{"key":"23_CR115","doi-asserted-by":"crossref","first-page":"1057","DOI":"10.1093\/brain\/awn025","volume":"131","author":"HD Rosas","year":"2008","unstructured":"Rosas HD, Salat DH, Lee SY et al (2008) Cerebral cortex and the clinical expression of Huntington\u2019s disease: complexity and heterogeneity. Brain 131:1057\u20131068","journal-title":"Brain"},{"key":"23_CR116","doi-asserted-by":"crossref","first-page":"977","DOI":"10.1002\/bies.201600048","volume":"38","author":"DC Rubinsztein","year":"2016","unstructured":"Rubinsztein DC, Orr HT (2016) Diminishing return for mechanistic therapeutics with neurodegenerative disease duration?: There may be a point in the course of a neurodegenerative condition where therapeutics targeting disease-causing mechanisms are futile. Bioessays 38:977\u2013980","journal-title":"Bioessays"}],"container-title":["Methods in Molecular Biology","Huntington\u2019s Disease"],"original-title":[],"link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/978-1-4939-7825-0_23","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,11,2]],"date-time":"2020-11-02T17:52:55Z","timestamp":1604339575000},"score":1,"resource":{"primary":{"URL":"http:\/\/link.springer.com\/10.1007\/978-1-4939-7825-0_23"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018]]},"ISBN":["9781493978243","9781493978250"],"references-count":116,"URL":"https:\/\/doi.org\/10.1007\/978-1-4939-7825-0_23","relation":{"is-part-of":[{"id-type":"doi","id":"https:\/\/doi.org\/10.1007\/978-1-4939-7825-0","asserted-by":"object"}]},"ISSN":["1064-3745","1940-6029"],"issn-type":[{"value":"1064-3745","type":"print"},{"value":"1940-6029","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018]]}}}