{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,2]],"date-time":"2026-11-02T08:52:30Z","timestamp":1793609550828,"version":"4.5.0"},"reference-count":44,"publisher":"National Academy of Sciences","issue":"41","content-domain":{"domain":["www.pnas.org"],"crossmark-restriction":true},"short-container-title":["Proc. Natl. Acad. Sci. U.S.A."],"published-print":{"date-parts":[[2011,10,11]]},"abstract":"<jats:p>\n                    Huntington disease (HD) is a progressive neurodegenerative disease that affects 30,000 individuals in North America. Treatments that slow its relentless course are not yet available, and biomarkers that can reliably measure disease activity and therapeutic response are urgently needed to facilitate their development. Here, we interrogated 119 human blood samples for transcripts associated with HD. We found that the dynamic regulator of chromatin plasticity\n                    <jats:italic>H2A histone family, member Y (H2AFY)<\/jats:italic>\n                    is specifically overexpressed in the blood and frontal cortex of patients with HD compared with controls. This association precedes the onset of clinical symptoms, was confirmed in two mouse models, and was independently replicated in cross-sectional and longitudinal clinical studies comprising 142 participants. A histone deacetylase inhibitor that suppresses neurodegeneration in animal models reduces\n                    <jats:italic>H2AFY<\/jats:italic>\n                    levels in a randomized phase II clinical trial. This study identifies the chromatin regulator\n                    <jats:italic>H2AFY<\/jats:italic>\n                    as a potential biomarker associated with disease activity and pharmacodynamic response that may become useful for enabling disease-modifying therapeutics for HD.\n                  <\/jats:p>","DOI":"10.1073\/pnas.1104409108","type":"journal-article","created":{"date-parts":[[2011,10,4]],"date-time":"2011-10-04T00:09:41Z","timestamp":1317686981000},"page":"17141-17146","update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":96,"title":["Transcriptional modulator\n                    <i>H2A histone family, member Y<\/i>\n                    (\n                    <i>H2AFY<\/i>\n                    ) marks Huntington disease activity in man and mouse"],"prefix":"10.1073","volume":"108","author":[{"given":"Yi","family":"Hu","sequence":"first","affiliation":[{"name":"Laboratory for Neurogenomics, Center for Neurologic Diseases, Harvard Medical School and Brigham and Women's Hospital, Cambridge, MA 02139;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vanita","family":"Chopra","sequence":"additional","affiliation":[{"name":"Laboratory of Neurodegeneration and Neurotherapeutics, MassGeneral Institute for Neurodegenerative Disease,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raman","family":"Chopra","sequence":"additional","affiliation":[{"name":"Laboratory of Neurodegeneration and Neurotherapeutics, MassGeneral Institute for Neurodegenerative Disease,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joseph J.","family":"Locascio","sequence":"additional","affiliation":[{"name":"Laboratory for Neurogenomics, Center for Neurologic Diseases, Harvard Medical School and Brigham and Women's Hospital, Cambridge, MA 02139;"},{"name":"Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02114;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhixiang","family":"Liao","sequence":"additional","affiliation":[{"name":"Laboratory for Neurogenomics, Center for Neurologic Diseases, Harvard Medical School and Brigham and Women's Hospital, Cambridge, MA 02139;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hongliu","family":"Ding","sequence":"additional","affiliation":[{"name":"Laboratory for Neurogenomics, Center for Neurologic Diseases, Harvard Medical School and Brigham and Women's Hospital, Cambridge, MA 02139;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bin","family":"Zheng","sequence":"additional","affiliation":[{"name":"Laboratory for Neurogenomics, Center for Neurologic Diseases, Harvard Medical School and Brigham and Women's Hospital, Cambridge, MA 02139;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wayne R.","family":"Matson","sequence":"additional","affiliation":[{"name":"Geriatric Research Education Clinical Center, New England Veterans Administration, Bedford, MA 01730;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert J.","family":"Ferrante","sequence":"additional","affiliation":[{"name":"Geriatric Research Educational and Clinical Center, Veterans Administration Pittsburgh Healthcare System, Pittsburgh, PA 15206;"},{"name":"Departments of Neurological Surgery, Neurology, and Neurobiology, University of Pittsburgh, Pittsburgh, PA 15213; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H. Diana","family":"Rosas","sequence":"additional","affiliation":[{"name":"Center for Neuroimaging of Aging and Neurodegenerative Diseases, and"},{"name":"Athinoula A. Martinos Center for Biomedical Imaging, Massachusetts General Hospital and Harvard Medical School, Charlestown, MA 02129;"},{"name":"Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02114;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven M.","family":"Hersch","sequence":"additional","affiliation":[{"name":"Laboratory of Neurodegeneration and Neurotherapeutics, MassGeneral Institute for Neurodegenerative Disease,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Clemens R.","family":"Scherzer","sequence":"additional","affiliation":[{"name":"Laboratory for Neurogenomics, Center for Neurologic Diseases, Harvard Medical School and Brigham and Women's Hospital, Cambridge, MA 02139;"},{"name":"Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02114;"},{"name":"Harvard NeuroDiscovery Center Biomarker Program, Cambridge, MA 02139"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"341","published-online":{"date-parts":[[2011,10,3]]},"reference":[{"key":"e_1_3_4_1_2","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1016\/S0168-9525(03)00074-X","article-title":"Transcriptional abnormalities in Huntington disease","volume":"19","author":"Sugars KL","year":"2003","unstructured":"KL Sugars, DC Rubinsztein, Transcriptional abnormalities in Huntington disease. 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Nat Neurosci 5, 731\u2013736 (2002).","journal-title":"Nat Neurosci"},{"key":"e_1_3_4_4_2","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1016\/j.neulet.2005.09.037","article-title":"The platelet maximum number of A2A-receptor binding sites (Bmax) linearly correlates with age at onset and CAG repeat expansion in Huntington's disease patients with predominant chorea","volume":"393","author":"Maglione V","year":"2006","unstructured":"V Maglione, et al., The platelet maximum number of A2A-receptor binding sites (Bmax) linearly correlates with age at onset and CAG repeat expansion in Huntington's disease patients with predominant chorea. Neurosci Lett 393, 27\u201330 (2006).","journal-title":"Neurosci Lett"},{"key":"e_1_3_4_5_2","doi-asserted-by":"crossref","first-page":"611","DOI":"10.1111\/j.1471-4159.2005.03070.x","article-title":"Tryptophan metabolism and oxidative stress in patients with Huntington's disease","volume":"93","author":"Stoy N","year":"2005","unstructured":"N Stoy, et al., Tryptophan metabolism and oxidative stress in patients with Huntington's disease. J Neurochem 93, 611\u2013623 (2005).","journal-title":"J Neurochem"},{"key":"e_1_3_4_6_2","doi-asserted-by":"crossref","first-page":"1911","DOI":"10.1093\/hmg\/11.17.1911","article-title":"Dysregulation of gene expression in the R6\/2 model of polyglutamine disease: Parallel changes in muscle and brain","volume":"11","author":"Luthi-Carter R","year":"2002","unstructured":"R Luthi-Carter, et al., Dysregulation of gene expression in the R6\/2 model of polyglutamine disease: Parallel changes in muscle and brain. 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Arch Neurol 61, 1200\u20131205 (2004).","journal-title":"Arch Neurol"},{"key":"e_1_3_4_12_2","doi-asserted-by":"crossref","first-page":"52ra73","DOI":"10.1126\/scitranslmed.3001059","article-title":"PGC-1\u03b1, a potential therapeutic target for early intervention in Parkinson's disease","volume":"2","author":"Zheng B","year":"2010","unstructured":"B Zheng, et al., PGC-1\u03b1, a potential therapeutic target for early intervention in Parkinson's disease. Sci Transl Med 2, 52ra73 (2010).","journal-title":"Sci Transl Med"},{"key":"e_1_3_4_13_2","doi-asserted-by":"crossref","first-page":"710","DOI":"10.1038\/ng1589","article-title":"An integrative genomics approach to infer causal associations between gene expression and disease","volume":"37","author":"Schadt EE","year":"2005","unstructured":"EE Schadt, et al., An integrative genomics approach to infer causal associations between gene expression and disease. 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