{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,21]],"date-time":"2026-09-21T09:24:43Z","timestamp":1789982683802,"version":"4.0.1"},"reference-count":78,"publisher":"Springer Science and Business Media LLC","issue":"3","license":[{"start":{"date-parts":[[2015,11,17]],"date-time":"2015-11-17T00:00:00Z","timestamp":1447718400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J Mol Neurosci"],"published-print":{"date-parts":[[2016,3]]},"DOI":"10.1007\/s12031-015-0676-5","type":"journal-article","created":{"date-parts":[[2015,11,17]],"date-time":"2015-11-17T05:34:59Z","timestamp":1447738499000},"page":"335-342","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Targeted Molecular Therapies for SBMA"],"prefix":"10.1007","volume":"58","author":[{"given":"Carlo","family":"Rinaldi","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bilal","family":"Malik","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda","family":"Greensmith","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2015,11,17]]},"reference":[{"key":"676_CR1","doi-asserted-by":"crossref","first-page":"2203","DOI":"10.1523\/JNEUROSCI.23-06-02203.2003","volume":"23","author":"H Adachi","year":"2003","unstructured":"Adachi H, Katsuno M, Minamiyama M, et al. (2003) Heat shock protein 70 chaperone overexpression ameliorates phenotypes of the spinal and bulbar muscular atrophy transgenic mouse model by reducing nuclear-localized mutant androgen receptor protein. J Neurosci. 23:2203\u20132211","journal-title":"J Neurosci."},{"key":"676_CR2","doi-asserted-by":"crossref","first-page":"659","DOI":"10.1093\/brain\/awh381","volume":"128","author":"H Adachi","year":"2005","unstructured":"Adachi H, Katsuno M, Minamiyama M, et al. (2005) Widespread nuclear and cytoplasmic accumulation of mutant androgen receptor in SBMA patients. Brain 128:659\u2013670","journal-title":"Brain"},{"key":"676_CR3","doi-asserted-by":"crossref","first-page":"1039","DOI":"10.1093\/hmg\/10.10.1039","volume":"10","author":"H Adachi","year":"2001","unstructured":"Adachi H, Kume A, Li M, et al. (2001) Transgenic mice with an expanded CAG repeat controlled by the human AR promoter show polyglutamine nuclear inclusions and neuronal dysfunction without neuronal cell death. Hum Mol Genet 10:1039\u20131048","journal-title":"Hum Mol Genet"},{"key":"676_CR4","doi-asserted-by":"crossref","first-page":"5115","DOI":"10.1523\/JNEUROSCI.1242-07.2007","volume":"27","author":"H Adachi","year":"2007","unstructured":"Adachi H, Waza M, Tokui K, et al. (2007) CHIP overexpression reduces mutant androgen receptor protein and ameliorates phenotypes of the spinal and bulbar muscular atrophy transgenic mouse model. J Neurosci. 27:5115\u20135126","journal-title":"J Neurosci."},{"issue":"14","key":"676_CR5","first-page":"61947","volume":"S0140-6736","author":"G Attard","year":"2015","unstructured":"Attard G, Parker C, Eeles RA, et al. (2015) Prostate cancer. Lancet pii: S0140-6736(14):61947\u201361944","journal-title":"Lancet pii:"},{"key":"676_CR6","doi-asserted-by":"crossref","first-page":"515","DOI":"10.1093\/hmg\/11.5.515","volume":"11","author":"CK Bailey","year":"2002","unstructured":"Bailey CK, Andriola IF, Kampinga HH, Merry DE (2002) Molecular chaperones enhance the degradation of expanded polyglutamine repeat androgen receptor in a cellular model of spinal and bulbar muscular atrophy. Hum Mol Genet 11:515\u2013523","journal-title":"Hum Mol Genet"},{"key":"676_CR7","doi-asserted-by":"crossref","first-page":"140","DOI":"10.1002\/ana.21540","volume":"65","author":"H Banno","year":"2009","unstructured":"Banno H, Katsuno M, Suzuki K (2009) Phase 2 trial of leuprorelin in patients with spinal and bulbar muscular atrophy. Ann Neurol 65:140\u2013150","journal-title":"Ann Neurol"},{"key":"676_CR8","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1016\/S1474-4422(10)70324-0","volume":"10","author":"H Banno","year":"2011","unstructured":"Banno H, Katsuno M, Suzuki K, Sobue G (2011) Dutasteride for spinal and bulbar muscular atrophy. Lancet Neurol 10:113\u2013115","journal-title":"Lancet Neurol"},{"key":"676_CR9","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1002\/mus.21310","volume":"40","author":"N Chahin","year":"2009","unstructured":"Chahin N, Sorenson EJ (2009) Serum creatine kinase levels in spinobulbar muscular atrophy and amyotrophic lateral sclerosis. Muscle Nerve 40:126\u2013129","journal-title":"Muscle Nerve"},{"key":"676_CR10","doi-asserted-by":"crossref","first-page":"4778","DOI":"10.1523\/JNEUROSCI.0808-04.2004","volume":"24","author":"ES Chevalier-Larsen","year":"2004","unstructured":"Chevalier-Larsen ES, O\u2019Brien CJ, Wang H, et al. (2004) Castration restores function and neurofilament alterations of aged symptomatic males in a transgenic mouse model of spinal and bulbar muscular atrophy. J Neurosci. 24:4778\u20134786","journal-title":"J Neurosci."},{"key":"676_CR11","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1172\/JCI73214","volume":"125","author":"JP Chua","year":"2015","unstructured":"Chua JP, Reddy SL, Yu Z, et al. (2015) Disrupting SUMOylation enhances transcriptional function and ameliorates polyglutamine androgen receptor-mediated disease. J Clin Invest 125:831\u2013845","journal-title":"J Clin Invest"},{"key":"676_CR12","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1016\/j.neuron.2014.03.001","volume":"82","author":"CJ Cortes","year":"2014","unstructured":"Cortes CJ, Ling SC, Guo LT, et al. (2014a) Muscle expression of mutant androgen receptor accounts for systemic and motor neuron disease phenotypes in spinal and bulbar muscular atrophy. Neuron 82:295\u2013307","journal-title":"Neuron"},{"key":"676_CR13","doi-asserted-by":"crossref","first-page":"1180","DOI":"10.1038\/nn.3787","volume":"17","author":"CJ Cortes","year":"2014","unstructured":"Cortes CJ, Miranda HC, Frankowski H, et al. (2014b) Polyglutamine-expanded androgen receptor interferes with TFEB to elicit autophagy defects in SBMA. Nat Neurosci 17:1180\u20131189","journal-title":"Nat Neurosci"},{"key":"676_CR14","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1002\/mus.21059","volume":"38","author":"ME Cudkowicz","year":"2008","unstructured":"Cudkowicz ME, Shefner JM, Simpson E, et al. (2008) Arimoclomol at dosages up to 300\u00a0mg\/day is well tolerated and safe in amyotrophic lateral sclerosis. Muscle Nerve 38:837\u2013844","journal-title":"Muscle Nerve"},{"key":"676_CR15","doi-asserted-by":"crossref","first-page":"896","DOI":"10.1038\/nature06783","volume":"452","author":"J Elm\u00e9n","year":"2008","unstructured":"Elm\u00e9n J, Lindow M, Sch\u00fctz S, et al. (2008) LNA-mediated microRNA silencing in non-human primates. Nature 452:896\u2013899","journal-title":"Nature"},{"key":"676_CR16","doi-asserted-by":"crossref","first-page":"375","DOI":"10.1016\/S0896-6273(03)00258-7","volume":"38","author":"ES Emamian","year":"2003","unstructured":"Emamian ES, Kaytor MD, Duvick LA, et al. (2003) Serine 776 of ataxin-1 is critical for polyglutamine-induced disease in SCA1 transgenic mice. Neuron 38:375\u2013387","journal-title":"Neuron"},{"key":"676_CR17","doi-asserted-by":"crossref","first-page":"140","DOI":"10.1016\/S1474-4422(10)70321-5","volume":"10","author":"LE Fern\u00e1ndez-Rhodes","year":"2011","unstructured":"Fern\u00e1ndez-Rhodes LE, Kokkinis AD, White MJ, et al. (2011) Efficacy and safety of dutasteride in patients with spinal and bulbar muscular atrophy: a randomised placebo-controlled trial. Lancet Neurol 10:140\u2013147","journal-title":"Lancet Neurol"},{"key":"676_CR18","doi-asserted-by":"crossref","first-page":"1519","DOI":"10.1038\/nn2011","volume":"10","author":"MJ Friedman","year":"2007","unstructured":"Friedman MJ, Shah AG, Fang ZH, et al. (2007) Polyglutamine domain modulates the TBP-TFIIB interaction: implications for its normal function and neurodegeneration. Nat Neurosci 10:1519\u20131528","journal-title":"Nat Neurosci"},{"key":"676_CR19","doi-asserted-by":"crossref","first-page":"1037","DOI":"10.1016\/j.drudis.2011.09.007","volume":"16","author":"I Gashaw","year":"2012","unstructured":"Gashaw I, Ellinghaus P, Sommer A, Asadullah K (2012) What makes a good drug target? Drug Discov Today 16:1037\u20131043","journal-title":"Drug Discov Today"},{"key":"676_CR20","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1093\/hmg\/ddu419","volume":"24","author":"E Giorgetti","year":"2015","unstructured":"Giorgetti E, Rusmini P, Crippa V, et al. (2015) Synergic prodegradative activity of bicalutamide and trehalose on the mutant androgen receptor responsible for spinal and bulbar muscular atrophy. Hum Mol Genet 24:64\u201375","journal-title":"Hum Mol Genet"},{"key":"676_CR21","first-page":"20","volume":"5","author":"J Huang","year":"2014","unstructured":"Huang J, Lyu H, Wang J, Liu B (2014) MicroRNA regulation and therapeutic targeting of survivin in cancer. Am J Cancer Res 5:20\u201331","journal-title":"Am J Cancer Res"},{"key":"676_CR22","doi-asserted-by":"crossref","first-page":"821","DOI":"10.1038\/nrd2132","volume":"5","author":"P Imming","year":"2006","unstructured":"Imming P, Sinning C, Meyer A (2006) Drugs, their targets and the nature and number of drug targets. Nat Rev Drug Discov. 5:821\u2013834","journal-title":"Nat Rev Drug Discov."},{"key":"676_CR23","doi-asserted-by":"crossref","first-page":"339","DOI":"10.1111\/j.1471-4159.2008.05595.x","volume":"107","author":"B Kalmar","year":"2008","unstructured":"Kalmar B, Novoselov S, Gray A, Cheetham ME, Margulis B, Greensmith L (2008) Late stage treatment with arimoclomol delays disease progression and prevents protein aggregation in the SOD1 mouse model of ALS. J Neurochem 107:339\u2013350","journal-title":"J Neurochem"},{"key":"676_CR24","doi-asserted-by":"crossref","first-page":"768","DOI":"10.1038\/nm878","volume":"9","author":"M Katsuno","year":"2003","unstructured":"Katsuno M, Adachi H, Doyu M, et al. (2003) Leuprorelin rescues polyglutamine-dependent phenotypes in a transgenic mouse model of spinal and bulbar muscular atrophy. Nat Med 9:768\u2013773","journal-title":"Nat Med"},{"key":"676_CR25","doi-asserted-by":"crossref","first-page":"843","DOI":"10.1016\/S0896-6273(02)00834-6","volume":"35","author":"M Katsuno","year":"2002","unstructured":"Katsuno M, Adachi H, Kume A, et al. (2002) Testosterone reduction prevents phenotypic expression in a transgenic mouse model of spinal and bulbar muscular atrophy. Neuron 35:843\u2013854","journal-title":"Neuron"},{"key":"676_CR26","doi-asserted-by":"crossref","first-page":"12106","DOI":"10.1523\/JNEUROSCI.3032-06.2006","volume":"26","author":"M Katsuno","year":"2006","unstructured":"Katsuno M, Adachi H, Minamiyama M, et al. (2006) Reversible disruption of dynactin 1-mediated retrograde axonal transport in polyglutamine-induced motor neuron degeneration. J Neurosci. 26:12106\u201312117","journal-title":"J Neurosci."},{"key":"676_CR27","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1016\/S1474-4422(10)70182-4","volume":"9","author":"M Katsuno","year":"2010","unstructured":"Katsuno M, Banno H, Suzuki K, et al. (2010) Efficacy and safety of leuprorelin in patients with spinal and bulbar muscular atrophy (JASMITT study): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Neurol 9:875\u2013884","journal-title":"Lancet Neurol"},{"key":"676_CR28","doi-asserted-by":"crossref","first-page":"16801","DOI":"10.1073\/pnas.0506249102","volume":"102","author":"M Katsuno","year":"2005","unstructured":"Katsuno M, Sang C, Adachi H, et al. (2005) Pharmacological induction of heat-shock proteins alleviates polyglutamine-mediated motor neuron disease. Proc Natl Acad Sci U S A 102:16801\u201316806","journal-title":"Proc Natl Acad Sci U S A"},{"key":"676_CR29","doi-asserted-by":"crossref","first-page":"246","DOI":"10.1016\/j.pneurobio.2012.05.007","volume":"99","author":"M Katsuno","year":"2012","unstructured":"Katsuno M, Tanaka F, Adachi H, et al. (2012) Pathogenesis and therapy of spinal and bulbar muscular atrophy (SBMA). Prog Neurobiol 99:246\u2013256","journal-title":"Prog Neurobiol"},{"key":"676_CR30","doi-asserted-by":"crossref","first-page":"588","DOI":"10.1038\/mt.2013.279","volume":"22","author":"MS Keiser","year":"2014","unstructured":"Keiser MS, Boudreau RL, Davidson BL (2014) Broad therapeutic benefit after RNAi expression vector delivery to deep cerebellar nuclei: implications for spinocerebellar ataxia type 1 therapy. Mol Ther 22:588\u2013595","journal-title":"Mol Ther"},{"key":"676_CR31","doi-asserted-by":"crossref","first-page":"402","DOI":"10.1038\/nm1021","volume":"10","author":"D Kieran","year":"2004","unstructured":"Kieran D, Kalmar B, Dick JR, Riddoch-Contreras J, Burnstock G, Greensmith L (2004) Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression in ALS mice. Nat Med 10:402\u2013405","journal-title":"Nat Med"},{"key":"676_CR32","first-page":"57","volume":"112","author":"JH Lee","year":"2005","unstructured":"Lee JH, Shin JH, Park KP, et al. (2005) Phenotypic variability in Kennedy\u2019s disease: implication of the early diagnostic features. Acta Neurol Scand 112:57\u201363","journal-title":"Acta Neurol Scand"},{"key":"676_CR33","doi-asserted-by":"crossref","first-page":"774","DOI":"10.1016\/j.celrep.2014.02.008","volume":"7","author":"AP Lieberman","year":"2014","unstructured":"Lieberman AP, Yu Z, Murray S, et al. (2014) Peripheral androgen receptor gene suppression rescues disease in mouse models of spinal and bulbar muscular atrophy. Cell Rep 7:774\u2013784","journal-title":"Cell Rep"},{"key":"676_CR34","doi-asserted-by":"crossref","first-page":"713","DOI":"10.1038\/nature06731","volume":"452","author":"J Lim","year":"2008","unstructured":"Lim J, Crespo-Barreto J, Jafar-Nejad P, et al. (2008) Opposing effects of polyglutamine expansion on native protein complexes contribute to SCA1. Nature 452:713\u2013718","journal-title":"Nature"},{"key":"676_CR35","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1002\/ana.410440216","volume":"44","author":"M Li","year":"1998","unstructured":"Li M, Miwa S, Kobayashi Y, et al. (1998) Nuclear inclusions of the androgen receptor protein in spinal and bulbar muscular atrophy. Ann Neurol 44:249\u2013254","journal-title":"Ann Neurol"},{"key":"676_CR36","doi-asserted-by":"crossref","first-page":"763","DOI":"10.1097\/01.bor.0000434671.77891.9a","volume":"25","author":"P Machado","year":"2013","unstructured":"Machado P, Brady S, Hanna MG (2013) Update in inclusion body myositis. Curr Opin Rheumatol 25:763\u2013771","journal-title":"Curr Opin Rheumatol"},{"key":"676_CR37","doi-asserted-by":"crossref","first-page":"1776","DOI":"10.1093\/hmg\/ddr061","volume":"20","author":"B Malik","year":"2011","unstructured":"Malik B, Nirmalananthan N, Bilsland LG, et al. (2011) Absence of disturbed axonal transport in spinal and bulbar muscular atrophy. Hum Mol Genet 20:1776\u20131786","journal-title":"Hum Mol Genet"},{"key":"676_CR38","doi-asserted-by":"crossref","first-page":"926","DOI":"10.1093\/brain\/aws343","volume":"136","author":"B Malik","year":"2013","unstructured":"Malik B, Nirmalananthan N, Gray AL, La Spada AR, Hanna MG, Greensmith L (2013) Co-induction of the heat shock response ameliorates disease progression in a mouse model of human spinal and bulbar muscular atrophy: implications for therapy. Brain 136:926\u2013943","journal-title":"Brain"},{"key":"676_CR39","doi-asserted-by":"crossref","first-page":"2197","DOI":"10.1093\/hmg\/9.14.2197","volume":"9","author":"A McCampbell","year":"2000","unstructured":"McCampbell A, Taylor JP, Taye AA, et al. (2000) CREB-binding protein sequestration by expanded polyglutamine. Hum Mol Genet 9:2197\u20132202","journal-title":"Hum Mol Genet"},{"key":"676_CR40","doi-asserted-by":"crossref","first-page":"162","DOI":"10.1016\/j.bcp.2013.08.006","volume":"87","author":"P McGonigle","year":"2014","unstructured":"McGonigle P, Ruggeri B (2014) Animal models of human disease: challenges in enabling translation. Biochem Pharmacol 87:162\u2013171","journal-title":"Biochem Pharmacol"},{"key":"676_CR41","doi-asserted-by":"crossref","first-page":"8478","DOI":"10.1073\/pnas.0503493102","volume":"102","author":"SJ McMahon","year":"2005","unstructured":"McMahon SJ, Pray-Grant MG, Schieltz D, Yates 3rd JR, Grant PA (2005) Polyglutamine-expanded spinocerebellar ataxia-7 protein disrupts normal SAGA and SLIK histone acetyltransferase activity. Proc Natl Acad Sci U S A 102:8478\u20138482","journal-title":"Proc Natl Acad Sci U S A"},{"key":"676_CR42","doi-asserted-by":"crossref","first-page":"1183","DOI":"10.1093\/hmg\/ddh131","volume":"13","author":"M Minamiyama","year":"2004","unstructured":"Minamiyama M, Katsuno M, Adachi H, et al. (2004) Sodium butyrate ameliorates phenotypic expression in a transgenic mouse model of spinal and bulbar muscular atrophy. Hum Mol Genet 13:1183\u20131192","journal-title":"Hum Mol Genet"},{"key":"676_CR43","doi-asserted-by":"crossref","first-page":"1136","DOI":"10.1038\/nm.2791","volume":"18","author":"Y Miyazaki","year":"2012","unstructured":"Miyazaki Y, Adachi H, Katsuno M, et al. (2012) Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2. Nat Med 18:1136\u20131141","journal-title":"Nat Med"},{"key":"676_CR44","doi-asserted-by":"crossref","first-page":"18259","DOI":"10.1073\/pnas.0705501104","volume":"104","author":"DA Monks","year":"2007","unstructured":"Monks DA, Johansen JA, Mo K, et al. (2007) Overexpression of wild-type androgen receptor in muscle recapitulates polyglutamine disease. Proc Natl Acad Sci U S A 104:18259\u201318264","journal-title":"Proc Natl Acad Sci U S A"},{"key":"676_CR45","doi-asserted-by":"crossref","first-page":"1894","DOI":"10.1093\/brain\/awu114","volume":"137","author":"K Montague","year":"2014","unstructured":"Montague K, Malik B, Gray AL, et al. (2014) Endoplasmic reticulum stress in spinal and bulbar muscular atrophy: a potential target for therapy. Brain 137:1894\u20131906","journal-title":"Brain"},{"key":"676_CR46","doi-asserted-by":"crossref","first-page":"1937","DOI":"10.1093\/hmg\/ddp115","volume":"18","author":"HL Montie","year":"2009","unstructured":"Montie HL, Cho MS, Holder L, et al. (2009) Cytoplasmic retention of polyglutamine-expanded androgen receptor ameliorates disease via autophagy in a mouse model of spinal and bulbar muscular atrophy. Hum Mol Genet 18:1937\u20131950","journal-title":"Hum Mol Genet"},{"key":"676_CR47","doi-asserted-by":"crossref","first-page":"17425","DOI":"10.1523\/JNEUROSCI.3958-11.2011","volume":"31","author":"HL Montie","year":"2011","unstructured":"Montie HL, Pestell RG, Merry DE (2011) SIRT1 modulates aggregation and toxicity through deacetylation of the androgen receptor in cell models of SBMA. J Neurosci. 31:17425\u201317436","journal-title":"J Neurosci."},{"key":"676_CR48","doi-asserted-by":"crossref","first-page":"907","DOI":"10.1038\/nn1717","volume":"9","author":"G Morfini","year":"2006","unstructured":"Morfini G, Pigino G, Szebenyi G, You Y, Pollema S, Brady ST (2006) JNK mediates pathogenic effects of polyglutamine-expanded androgen receptor on fast axonal transport. Nat Neurosci 9:907\u2013916","journal-title":"Nat Neurosci"},{"key":"676_CR49","doi-asserted-by":"crossref","first-page":"936","DOI":"10.1016\/j.neuron.2010.08.034","volume":"67","author":"NB Nedelsky","year":"2010","unstructured":"Nedelsky NB, Pennuto M, Smith RB, et al. (2010) Native functions of the androgen receptor are essential to pathogenesis in a drosophila model of spinobulbar muscular atrophy. Neuron 67:936\u2013952","journal-title":"Neuron"},{"key":"676_CR50","doi-asserted-by":"crossref","first-page":"35567","DOI":"10.1074\/jbc.M110.146845","volume":"285","author":"CR Orr","year":"2010","unstructured":"Orr CR, Montie HL, Liu Y, et al. (2010) An interdomain interaction of the androgen receptor is required for its aggregation and toxicity in spinal and bulbar muscular atrophy. J Biol Chem. 285:35567\u201333577","journal-title":"J Biol Chem."},{"key":"676_CR51","doi-asserted-by":"crossref","first-page":"1593","DOI":"10.1093\/hmg\/ddm109","volume":"16","author":"I Palazzolo","year":"2007","unstructured":"Palazzolo I, Burnett BG, Young JE, et al. (2007) Akt blocks ligand binding and protects against expanded polyglutamine androgen receptor toxicity. Hum Mol Genet 16:1593\u20131603","journal-title":"Hum Mol Genet"},{"key":"676_CR52","doi-asserted-by":"crossref","first-page":"316","DOI":"10.1016\/j.neuron.2009.07.019","volume":"63","author":"I Palazzolo","year":"2009","unstructured":"Palazzolo I, Stack C, Kong L, et al. (2009) Overexpression of IGF-1 in muscle attenuates disease in a mouse model of spinal and bulbar muscular atrophy. Neuron 63:316\u2013328","journal-title":"Neuron"},{"key":"676_CR53","doi-asserted-by":"crossref","first-page":"8472","DOI":"10.1073\/pnas.0503505102","volume":"102","author":"VB Palhan","year":"2005","unstructured":"Palhan VB, Chen S, Peng GH, et al. (2005) Polyglutamine-expanded ataxin-7 inhibits STAGA histone acetyltransferase activity to produce retinal degeneration. Proc Natl Acad Sci U S A 102:8472\u20138477","journal-title":"Proc Natl Acad Sci U S A"},{"key":"676_CR54","doi-asserted-by":"crossref","first-page":"859","DOI":"10.1038\/nature05853","volume":"447","author":"UB Pandey","year":"2007","unstructured":"Pandey UB, Nie Z, Batlevi Y, et al. (2007) HDAC6 rescues neurodegeneration and provides an essential link between autophagy and the UPS. Nature 447:859\u2013863","journal-title":"Nature"},{"key":"676_CR55","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1093\/hmg\/ddn310","volume":"18","author":"S Ranganathan","year":"2009","unstructured":"Ranganathan S, Harmison GG, Meyertholen K, Pennuto M, Burnett BG, Fischbeck KH (2009) Mitochondrial abnormalities in spinal and bulbar muscular atrophy. Hum Mol Genet 18:27\u201342","journal-title":"Hum Mol Genet"},{"key":"676_CR56","doi-asserted-by":"crossref","first-page":"1261","DOI":"10.2119\/molmed.2012.00271","volume":"18","author":"C Rinaldi","year":"2012","unstructured":"Rinaldi C, Bott LC, Chen KL, et al. (2012) Insulinlike growth factor (IGF)-1 administration ameliorates disease manifestations in a mouse model of spinal and bulbar muscular atrophy. Mol Med 18:1261\u20131268","journal-title":"Mol Med"},{"key":"676_CR57","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1016\/j.neuron.2014.04.005","volume":"82","author":"C Rinaldi","year":"2014","unstructured":"Rinaldi C, Bott LC, Fischbeck KH (2014) Muscle matters in Kennedy\u2019s disease. Neuron 82:251\u2013253","journal-title":"Neuron"},{"key":"676_CR58","doi-asserted-by":"crossref","first-page":"1909","DOI":"10.1038\/mt.2013.152","volume":"21","author":"E Rodr\u00edguez-Lebr\u00f3n","year":"2013","unstructured":"Rodr\u00edguez-Lebr\u00f3n E, Costa Mdo C, Luna-Cancalon K, et al. (2013) Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice. Mol Ther 21:1909\u20131918","journal-title":"Mol Ther"},{"key":"676_CR59","doi-asserted-by":"crossref","first-page":"361","DOI":"10.1016\/j.nbd.2010.06.016","volume":"40","author":"P Rusmini","year":"2010","unstructured":"Rusmini P, Bolzoni E, Crippa V, et al. (2010) Proteasomal and autophagic degradative activities in spinal and bulbar muscular atrophy. Neurobiol Dis 40:361\u2013369","journal-title":"Neurobiol Dis"},{"key":"676_CR60","doi-asserted-by":"crossref","first-page":"2585","DOI":"10.1016\/j.neurobiolaging.2013.05.026","volume":"34","author":"P Rusmini","year":"2013","unstructured":"Rusmini P, Crippa V, Giorgetti E, et al. (2013) Clearance of the mutant androgen receptor in motoneuronal models of spinal and bulbar muscular atrophy. Neurobiol Aging 34:2585\u20132603","journal-title":"Neurobiol Aging"},{"key":"676_CR61","doi-asserted-by":"crossref","unstructured":"Sahashi K, Katsuno M, Hung G, et al. (2015) Silencing neuronal mutant androgen receptor in a mouse model of spinal and bulbar muscular atrophy. Hum Mol Genet. pii: ddv300.","DOI":"10.1093\/hmg\/ddv300"},{"key":"676_CR62","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1093\/hmg\/9.1.133","volume":"9","author":"S Simeoni","year":"2000","unstructured":"Simeoni S, Mancini MA, Stenoien DL, et al. (2000) Motoneuronal cell death is not correlated with aggregate formation of androgen receptors containing an elongated polyglutamine tract. Hum Mol Genet 9:133\u2013144","journal-title":"Hum Mol Genet"},{"key":"676_CR63","doi-asserted-by":"crossref","first-page":"209","DOI":"10.1093\/brain\/112.1.209","volume":"112","author":"G Sobue","year":"1989","unstructured":"Sobue G, Hashizume Y, Mukai E, Hirayama M, Mitsuma T, Takahashi A (1989) X-linked recessive bulbospinal neuronopathy. A clinicopathological study. Brain 112:209\u2013232","journal-title":"Brain"},{"key":"676_CR64","doi-asserted-by":"crossref","first-page":"287","DOI":"10.1080\/17482960802206801","volume":"9","author":"G Sorar\u00f9","year":"2008","unstructured":"Sorar\u00f9 G, D\u2019Ascenzo C, Nicolao P, et al. (2008) Muscle histopathology in upper motor neuron-dominant amyotrophic lateral sclerosis. Amyotroph Lateral Scler 9:287\u2013293","journal-title":"Amyotroph Lateral Scler"},{"key":"676_CR65","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1093\/hmg\/8.5.731","volume":"8","author":"DL Stenoien","year":"1999","unstructured":"Stenoien DL, Cummings CJ, Adams HP, et al. (1999) Polyglutamine-expanded androgen receptors form aggregates that sequester heat shock proteins, proteasome components and SRC-1, and are suppressed by the HDJ-2 chaperone. Hum Mol Genet 8:731\u2013741","journal-title":"Hum Mol Genet"},{"key":"676_CR66","doi-asserted-by":"crossref","first-page":"855","DOI":"10.1016\/S0896-6273(02)00875-9","volume":"35","author":"K Takeyama","year":"2002","unstructured":"Takeyama K, Ito S, Yamamoto A, et al. (2002) Androgen-dependent neurodegeneration by polyglutamine-expanded human androgen receptor in drosophila. Neuron 35:855\u2013864","journal-title":"Neuron"},{"key":"676_CR67","doi-asserted-by":"crossref","first-page":"3552","DOI":"10.1093\/hmg\/ddu066","volume":"23","author":"G Tohnai","year":"2014","unstructured":"Tohnai G, Adachi H, Katsuno M, et al. (2014) Paeoniflorin eliminates a mutant AR via NF-YA-dependent proteolysis in spinal and bulbar muscular atrophy. Hum Mol Genet 23:3552\u20133565","journal-title":"Hum Mol Genet"},{"key":"676_CR68","doi-asserted-by":"crossref","first-page":"898","DOI":"10.1093\/hmg\/ddn419","volume":"18","author":"K Tokui","year":"2009","unstructured":"Tokui K, Adachi H, Waza M, et al. (2009) 17-DMAG ameliorates polyglutamine-mediated motor neuron degeneration through well-preserved proteasome function in an SBMA model mouse. Hum Mol Genet 18:898\u2013910","journal-title":"Hum Mol Genet"},{"key":"676_CR69","doi-asserted-by":"crossref","first-page":"1150","DOI":"10.1038\/nm1097-1150","volume":"3","author":"L Vigh","year":"1997","unstructured":"Vigh L, Literati PN, Horvath I, et al. (1997) Bimoclomol: a nontoxic, hydroxylamine derivative with stress protein-inducing activity and cytoprotective effects. Nat Med 3:1150\u20131154","journal-title":"Nat Med"},{"key":"676_CR70","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1038\/nchembio.1140","volume":"9","author":"AM Wang","year":"2013","unstructured":"Wang AM, Miyata Y, Klinedinst S, et al. (2013) Activation of Hsp70 reduces neurotoxicity by promoting polyglutamine protein degradation. Nat Chem Biol 9:112\u2013118","journal-title":"Nat Chem Biol"},{"key":"676_CR71","doi-asserted-by":"crossref","first-page":"1088","DOI":"10.1038\/nm1298","volume":"11","author":"M Waza","year":"2005","unstructured":"Waza M, Adachi H, Katsuno M, et al. (2005) 17-AAG, an Hsp90 inhibitor, ameliorates polyglutamine-mediated motor neuron degeneration. Nat Med 11:1088\u20131095","journal-title":"Nat Med"},{"key":"676_CR72","doi-asserted-by":"crossref","DOI":"10.1017\/erm.2011.1","volume":"14","author":"LM Watson","year":"2012","unstructured":"Watson LM, Wood MJ (2012) RNA therapy for polyglutamine neurodegenerative diseases. Expert Rev Mol Med 14:e3","journal-title":"Expert Rev Mol Med"},{"key":"676_CR73","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1038\/nrd2898","volume":"8","author":"M. Wehling","year":"2009","unstructured":"Wehling M (2009) Assessing the translatability of drug projects: what needs to be scored to predict success? Nat Rev Drug Discov. 8:541\u2013546","journal-title":"Nat Rev Drug Discov."},{"key":"676_CR74","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1038\/nm1547","volume":"13","author":"Z Yang","year":"2007","unstructured":"Yang Z, Chang YJ, Yu IC, et al. (2007) ASC-J9 ameliorates spinal and bulbar muscular atrophy phenotype via degradation of androgen receptor. Nat Med 13:348\u2013353","journal-title":"Nat Med"},{"key":"676_CR75","doi-asserted-by":"crossref","first-page":"2663","DOI":"10.1172\/JCI28773","volume":"116","author":"Z Yu","year":"2006","unstructured":"Yu Z, Dadgar N, Albertelli M, et al. (2006) Androgen-dependent pathology demonstrates myopathic contribution to the Kennedy disease phenotype in a mouse knock-in model. J Clin Invest 116:2663\u20132672","journal-title":"J Clin Invest"},{"key":"676_CR76","volume":"7","author":"Z Yu","year":"2011","unstructured":"Yu Z, Wang AM, Adachi H, et al. (2011) Macroautophagy is regulated by the UPR-mediator CHOP and accentuates the phenotype of SBMA mice. PLoS Genet 7:e1002321","journal-title":"PLoS Genet"},{"key":"676_CR77","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1124\/pr.58.2.4","volume":"58","author":"CJ Zheng","year":"2006","unstructured":"Zheng CJ, Han LY, Yap CW, Ji ZL, Cao ZW, Chen YZ (2006) Therapeutic targets: progress of their exploration and investigation of their characteristics. Pharmacol Rev 58:259\u2013279","journal-title":"Pharmacol Rev"},{"key":"676_CR78","doi-asserted-by":"crossref","first-page":"7425","DOI":"10.1074\/jbc.R800041200","volume":"284","author":"HY Zoghbi","year":"2009","unstructured":"Zoghbi HY, Orr HT (2009) Pathogenic mechanisms of a polyglutamine-mediated neurodegenerative disease, spinocerebellar ataxia type 1. J Biol Chem. 284:7425\u20137429","journal-title":"J Biol Chem."}],"container-title":["Journal of Molecular Neuroscience"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s12031-015-0676-5.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/article\/10.1007\/s12031-015-0676-5\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s12031-015-0676-5","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,9,1]],"date-time":"2019-09-01T16:02:29Z","timestamp":1567353749000},"score":1,"resource":{"primary":{"URL":"http:\/\/link.springer.com\/10.1007\/s12031-015-0676-5"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2015,11,17]]},"references-count":78,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2016,3]]}},"alternative-id":["676"],"URL":"https:\/\/doi.org\/10.1007\/s12031-015-0676-5","relation":{},"ISSN":["0895-8696","1559-1166"],"issn-type":[{"value":"0895-8696","type":"print"},{"value":"1559-1166","type":"electronic"}],"subject":[],"published":{"date-parts":[[2015,11,17]]}}}