Gitler AD, Dhillon P, Shorter J. Neurodegenerative disease: models, mechanisms, and a new hope. Dis Model Mech 2017; 10: 499-502.
Armakola M, Higgins MJ, Figley MD, et al. Inhibition of RNA lariat debranching enzyme suppresses TDP-43 toxicity in ALS disease models. Nat Genet 2012; 44: 1302-9.
Becker LA, Huang B, Bieri G, et al. Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice. Nature 2017; 544: 367-71.
Chaudhary RK, Kardani J, Singh K, et al. Deciphering the roles of trehalose and Hsp104 in the inhibition of aggregation of mutant huntingtin in a yeast model of Huntington’s disease. Neuromolecular Med 2014; 16: 280-91.
Sarkar S, Perlstein EO, Imarisio S, et al. Small molecules enhance autophagy and reduce toxicity in Huntington’s disease models. Nat Chem Biol 2007; 3: 331-8.
Williams A, Sarkar S, Cuddon P, et al. Novel targets for Huntington’s disease in an mTOR-independent autophagy pathway. Nat Chem Biol 2008; 4: 295-305.
Balchin D, Hayer-Hartl M, Hartl FU. In vivo aspects of protein folding and quality control. Science 2016; 353aac4354
Carroll JB, Warby SC, Southwell AL, et al. Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin. Mol Ther 2011; 19: 2178-85.
Zielonka D, Mielcarek M, Landwehrmeyer GB. Update on Huntington’s disease: advances in care and emerging therapeutic options. Parkinsonism Relat Disord 2015; 21: 169-78.
Labbadia J, Morimoto RI. The biology of proteostasis in aging and disease. Annu Rev Biochem 2015; 84: 435-64.
Sweeney P, Park H, Baumann M, et al. Protein misfolding in neurodegenerative diseases: implications and strategies. Transl Neurodegener 2017; 6: 6.
Finka A, Sharma SK, Goloubinoff P. Multi-layered molecular mechanisms of polypeptide holding, unfolding and disaggregation by HSP70/HSP110 chaperones. Front Mol Biosci 2015; 2: 29.
Hipp MS, Park S-H, Hartl FU. Proteostasis impairment in protein-misfolding and-aggregation diseases. Trends Cell Biol 2014; 24: 506-14.
Goedert M, Spillantini MG, Del TK, et al. 100 years of Lewy pathology. Nat Rev Neurol 2013; 9: 13-24.
Bates GP, Dorsey R, Gusella JF, et al. Huntington’s disease. Nat Rev Dis Primers 2015; 1: 1-21.
Williams AJ, Paulson HL. Polyglutamine neurodegeneration: protein misfolding revisited. Trends Neurosci 2008; 31: 521-8.
Mattson MP. Pathways towards and away from Alzheimer’s disease. Nature 2004; 430: 631-9.
Ajroud-Driss S, Siddique T. Sporadic and hereditary amyotrophic lateral sclerosis (ALS). Biochim Biophys Acta 2015; 1852: 679-84.
Geschwind MD. Prion diseases. Continuum (Minneap Minn) 2015; 21: 1612-38.
Kampinga HH, Bergink S. Heat shock proteins as potential targets for protective strategies in neurodegeneration. Lancet Neurol 2016; 15: 748-59.
Tanaka M, Machida Y, Niu S, et al. Trehalose alleviates polyglutamine-mediated pathology in a mouse model of Huntington disease. Nat Med 2004; 10: 148-54.
Finkbeiner S. Huntington’s disease. Cold Spring Harb Perspect Biol 2011; 3a007476
Ross CA, Aylward EH, Wild EJ, et al. Huntington disease: natural history, biomarkers and prospects for therapeutics. Nat Rev Neurol 2014; 10: 204-16.
Imarisio S, Carmichael J, Korolchuk V, et al. Huntington’s disease: from pathology and genetics to potential therapies. Biochem J 2008; 412: 191-209.
Landles C, Bates GP. Huntingtin and the molecular pathogenesis of Huntington’s disease. EMBO Rep 2004; 5: 958-63.
Baig SS, Strong M, Quarrell OW. The global prevalence of Huntington’s disease: a systematic review and discussion. Neurodegener Dis Manag 2016; 6: 331-43.
Fisher ER, Hayden MR. Multisource ascertainment of Huntington disease in Canada: prevalence and population at risk. Mov Disord 2013; 29: 105-14.
Pramanik S, Basu P, Gangopadhaya PK, et al. Analysis of CAG and CCG repeats in Huntingtin gene among HD patients and normal populations of India. Eur J Hum Genet 2000; 8: 678-82.
Xu M, Wu ZY. Huntington disease in Asia. Chin Med J (Engl) 2015; 128: 1815-9.
Estrada-Sanchez AM, Rebec GV. Role of cerebral cortex in the neuropathology of Huntington’s disease. Front Neural Circuits 2013; 7: 1-9.
Dickey AS, La Spada AR. Therapy development in Huntington disease: From current strategies to emerging opportunities. Am J Med Genet A 2018; 176: 842-61.
Schwab LC, Garas SN, Drouin-Ouellet J, et al. Dopamine and Huntington’s disease. Expert Rev Neurother 2015; 15: 445-58.
Yero T, Rey JA. Tetrabenazine (Xenazine), an FDA-approved treatment option for huntington’s disease-related chorea. P&T 2008; 33: 690-4.
Frank S. Treatment of Huntington’s disease. Neurotherapeutics 2014; 11: 153-60.
Paleacu D, Anca M, Giladi N. Olanzapine in Huntington’s disease. Acta Neurol Scand 2002; 105: 441-4.
Patzold T, Brune M. Obsessive compulsive disorder in huntington disease: a case of isolated obsessions successfully treated with sertraline. Neuropsychiatry Neuropsychol Behav Neurol 2002; 15: 216-9.
Madhusoodanan S, Brenner R. Use of risperidone in psychosis associated with Huntington’s disease. Am J Geriatr Psychiatry 1998; 6: 347-9.
Saft C, Andrich J, Kraus PH, et al. Amisulpride in Huntington’s disease. Psychiatr Prax 2005; 32: 363-6.
Claassen DO, Carroll B, De Boer LM, et al. Indirect tolerability comparison of Deutetrabenazine and Tetrabenazine for Huntington disease. J Clin Mov Disord 2017; 4: 3.
Kieburtz K, Reilmann R, Olanow CW. Huntington’s disease: current and future therapeutic prospects. Mov Disord 2018; 33: 1033-41.
Rodrigues FB, Duarte GS, Costa J, et al. Tetrabenazine versus Deutetrabenazine for Huntington’s disease: twins or distant cousins? Mov Disord Clin Pract 2017; 4: 582-5.
Vacher C, Garcia-Oroz L, Rubinsztein DC. Overexpression of yeast hsp104 reduces polyglutamine aggregation and prolongs survival of a transgenic mouse model of Huntington’s disease. Hum Mol Genet 2005; 14: 3425-33.
Wyttenbach A, Sauvageot O, Carmichael J, et al. Heat shock protein 27 prevents cellular polyglutamine toxicity and suppresses the increase of reactive oxygen species caused by huntingtin. Hum Mol Genet 2002; 11: 1137-51.
Chan HYE, Warrick JM, Gray-Board GL, et al. Mechanisms of chaperone suppression of polyglutamine disease: selectivity, synergy and modulation of protein solubility in Drosophila. Hum Mol Genet 2000; 9: 2811-20.
Warrick JM, Chan HY, Gray-Board GL, et al. Suppression of polyglutamine-mediated neurodegeneration in Drosophila by the molecular chaperone HSP70. Nat Genet 1999; 23: 425-8.
Satyal SH, Schmidt E, Kitagawa K, et al. Polyglutamine aggregates alter protein folding homeostasis in Caenorhabditis elegans. Proc Natl Acad Sci USA 2000; 97: 5750-5.
Meriin AB, Zhang X, He X, et al. Huntingtin toxicity in yeast model depends on polyglutamine aggregation mediated by a prion-like protein Rnq1. J Cell Biol 2002; 157: 997-1004.
Saleh AA, Gune US, Chaudhary RK, et al. Roles of Hsp104 and trehalose in solubilisation of mutant huntingtin in heat shocked Saccharomyces cerevisiae cells. Biochim Biophys Acta 2014; 1843: 746-57.
Perrin VR, Régulier E, Abbas-Terki T, et al. Neuroprotection by Hsp104 and Hsp27 in lentiviral-based rat models of Huntington’s disease. Mol Ther 2007; 15: 903-11.
Carmichael J, Chatellier J, Woolfson A, et al. Bacterial and yeast chaperones reduce both aggregate formation and cell death in mammalian cell models of Huntington’s disease. Proc Natl Acad Sci USA 2000; 97: 9701-5.
Sittler A, Lurz R, Lueder G, et al. Geldanamycin activates a heat shock response and inhibits huntingtin aggregation in a cell culture model of Huntington’s disease. Hum Mol Genet 2001; 10: 1307-15.
Samuni Y, Ishii H, Hyodo F, et al. Reactive oxygen species mediate hepatotoxicity induced by the Hsp90 inhibitor geldanamycin and its analogs. Free Radic Biol Med 2010; 48: 1559-63.
Hay DG, Sathasivam K, Tobaben S, et al. Progressive decrease in chaperone protein levels in a mouse model of Huntington’s disease and induction of stress proteins as a therapeutic approach. Hum Mol Genet 2004; 13: 1389-405.
Calamini B, Silva MC, Madoux F, et al. Small-molecule proteostasis regulators for protein conformational diseases. Nat Chem Biol 2011; 8: 185-96.
Neef DW, Turski ML, Thiele DJ. Modulation of heat shock transcription factor 1 as a therapeutic target for small molecule intervention in neurodegenerative disease. PLoS Biol 2010; 8e1000291
Tsaytler P, Harding HP, Ron D, et al. Selective inhibition of a regulatory subunit of protein phosphatase 1 restores proteostasis. Science 2011; 332: 91-4.
Das I, Krzyzosiak A, Schneider K, et al. Preventing proteostasis diseases by selective inhibition of a phosphatase regulatory subunit. Science 2015; 348: 239-42.
Ravikumar B, Vacher C, Berger Z, et al. Inhibition of mTOR induces autophagy and reduces toxicity of polyglutamine expansions in fly and mouse models of Huntington disease. Nat Genet 2004; 36: 585-95.
Sarkar S, Davies JE, Huang Z, et al. Trehalose, a novel mTOR-independent autophagy enhancer, accelerates the clearance of mutant huntingtin and α-synuclein. J Biol Chem 2007; 282: 5641-52.
Marelli C, Maschat F. The P42 peptide and peptide-based therapies for Huntington’s disease. Orphanet J Rare Dis 2016; 11: 24.
Maschat F, Parmentier M-L, Bonneaud L, et al. Therapeutic peptides and use thereof against
Huntington's disease. US8987211. 2015.
Burra G, Thakur AK. Inhibition of polyglutamine aggregation by SIMILAR huntingtin N-terminal sequences: Prospective molecules for preclinical evaluation in Huntington’s disease. Biopolymers 2017; 108e23021
Zhang Q, Chen ZS, An Y, et al. A peptidylic inhibitor for neutralizing expanded CAG RNA-induced nucleolar stress in polyglutamine diseases. RNA 2018; 24: 486.
Vagenende V, Yap MGS, Trout BL. Mechanisms of protein stabilization and prevention of protein aggregation by glycerol. Biochemistry 2009; 48: 11084-96.
Harper SQ, Staber PD, He X, et al. RNA interference improves motor and neuropathological abnormalities in a Huntington’s disease mouse model. Proc Natl Acad Sci USA 2005; 102: 5820-5.
Rodriguez-Lebron E, Denovan-Wright EM, Nash K, et al. Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington’s disease transgenic mice. Mol Ther 2005; 12: 618-33.
Wang YL, Liu W, Wada E, et al. Clinico-pathological rescue of a model mouse of Huntington’s disease by siRNA. Neurosci Res 2005; 53: 241-9.
DiFiglia M, Sena-Esteves M, Chase K, et al. Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits. Proc Natl Acad Sci USA 2007; 104: 17204-9.
Stanek LM, Sardi SP, Mastis B, et al. Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington’s disease. Hum Gene Ther 2014; 25: 461-74.
Becanovic K, Norremolle A, Neal SJ, et al. A SNP in the HTT promoter alters NF-kappaB binding and is a bidirectional genetic modifier of Huntington disease. Nat Neurosci 2015; 18: 807-16.
Hu J, Matsui M, Gagnon KT, et al. Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs. Nat Biotechnol 2009; 27: 478-84.
Monteys AM, Wilson MJ, Boudreau RL, et al. Artificial miRNAs targeting mutant huntingtin show preferential silencing in vitro and in vivo. Mol Ther Nucleic Acids 2015; 4e234
Kordasiewicz HB, Stanek LM, Wancewicz EV, et al. Sustained therapeutic reversal of Huntington’s disease by transient repression of huntingtin synthesis. Neuron 2012; 74: 1031-44.
Southwell AL, Skotte NH, Kordasiewicz HB, et al. In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides. Mol Ther 2014; 22: 2093-106.
Caron NS, Dorsey ER, Hayden MR. Therapeutic approaches to Huntington disease: from the bench to the clinic. Nat Rev Drug Discov 2018; 17: 729-50.
Kay C, Collins JA, Skotte NH, et al. Huntingtin haplotypes provide prioritized target panels for allele-specific silencing in Huntington Disease patients of European ancestry. Mol Ther 2015; 23: 1759-71.
Zhou J, Rossi J. Aptamers as targeted therapeutics: current potential and challenges. Nat Rev Drug Discov 2017; 16: 440.
Nimjee SM, White RR, Becker RC, et al. Aptamers as therapeutics. Annu Rev Pharmacol Toxicol 2017; 57: 61-79.
Chaudhary RK, Patel KA, Patel MK, et al. Inhibition of aggregation of mutant huntingtin by nucleic acid aptamers in vitro and in a yeast model of Huntington’s disease. Mol Ther 2015; 23: 1912-26.
Shin B, Jung R, Oh H, et al. Novel DNA aptamers that bind to mutant huntingtin and modify its activity. Mol Ther Nucleic Acids 2018; 11: 416-28.
Skogen M, Roth J, Yerkes S, et al. Short G-rich oligonucleotides as a potential therapeutic for Huntington’s Disease. BMC Neurosci 2006; 7: 65.
Tsukakoshi K, Harada R, Sode K, et al. Screening of DNA aptamer which binds to alpha-synuclein. Biotechnol Lett 2010; 32: 643-8.
Rahimi F, Murakami K, Summers JL, et al. RNA aptamers generated against oligomeric Abeta40 recognize common amyloid aptatopes with low specificity but high sensitivity. PLoS One 2009; 4e7694
Rhie A, Kirby L, Sayer N, et al. Characterization of 2′-fluoro-RNA aptamers that bind preferentially to disease-associated conformations of prion protein and inhibit conversion. J Biol Chem 2003; 278: 39697-705.
Patel KP, Chaudhary RK, Roy I. RNA aptamers rescue mitochondrial dysfunction in a yeast model of Huntington’s disease. Mol Ther Nucleic Acids 2018; 12: 45-56.
Hands S, Sajjad MU, Newton MJ, et al. In vitro and in vivo aggregation of a fragment of huntingtin protein directly causes free radical production. J Biol Chem 2011; 286: 44512-20.
Patel KA, Kolluri T, Jain S, et al. Designing aptamers which respond to intracellular oxidative stress and inhibit aggregation of mutant huntingtin. Free Radic Biol Med 2018; 120: 311-6.
Drouet V, Perrin V, Hassig R, et al. Sustained effects of nonallele-specific Huntingtin silencing. Ann Neurol 2009; 65: 276-85.
Huang B, Schiefer J, Sass C, et al. High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo. Hum Gene Ther 2007; 18: 303-11.
Ramaswamy S, Kordower JH. Gene therapy for Huntington’s disease. Neurobiol Dis 2012; 48: 243-54.
Zincarelli C, Soltys S, Rengo G, Rabinowitz JE. Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 2008; 16: 1073-80.
McBride JL, Pitzer MR, Boudreau RL, et al. Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington’s disease. Mol Ther 2011; 19: 2152-62.
Nayak S, Herzog RW. Progress and prospects: immune responses to viral vectors. Gene Ther 2010; 17: 295-304.
Godinho BM, Ogier JR, Darcy R, et al. Self-assembling modified beta-cyclodextrin nanoparticles as neuronal siRNA delivery vectors: focus on Huntington’s disease. Mol Pharm 2013; 10: 640-9.
Stiles DK, Zhang Z, Ge P, et al. Widespread suppression of huntingtin with convection-enhanced delivery of siRNA. Exp Neurol 2012; 233: 463-71.
Alvarez-Erviti L, Seow Y, Yin H, et al. Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes. Nat Biotechnol 2011; 29: 341-5.
Keiser MS, Kordasiewicz HB, McBride JL. Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington’s disease and spinocerebellar ataxia. Hum Mol Genet 2016; 25: R53-64.
Wild EJ, Tabrizi SJ. Therapies targeting DNA and RNA in Huntington’s disease. Lancet Neurol 2017; 16: 837-47.
Tan JY, Sellers DL, Pham B, et al. Non-viral nucleic acid delivery strategies to the central nervous system. Front Mol Neurosci 2016; 9: 108.
Yu D, Pendergraff H, Liu J, et al. Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression. Cell 2012; 150: 895-908.
Chiriboga CA, Swoboda KJ, Darras BT, et al. Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy. Neurology 2016; 86: 890-7.
Hache M, Swoboda KJ, Sethna N, et al. Intrathecal injections in children with spinal muscular atrophy: Nusinersen clinical trial experience. J Child Neurol 2016; 31: 899-906.
Leavitt B, Tabrizi S, Kordasiewicz H, et al. Discovery
and early clinical development of ISIS-HTTRx,
the first HTT-lowering drug to be tested in patients
with Huntington's disease (PL01.002). Neurology
2016; 86: PL01.002.
Miller TM, Pestronk A, David W, et al. 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 2013; 12: 435-42.
Tabrizi S, Leavitt B, Kordasiewicz H, et al. Effects of
IONIS-HTTRx in patients with early Huntington's
Disease, results of the first HTT-lowering drug trial
(CT.002). Neurology 2018; 90: CT.002.
Aartsma-Rus A. FDA Approval of Nusinersen for spinal muscular atrophy makes 2016 the year of splice modulating oligonucleotides. Nucleic Acid Ther 2017; 27: 67-9.
Finkel RS, Chiriboga CA, Vajsar J, et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet 2016; 388: 3017-26.
Huntington Study Group. Effect of deutetrabenazine on chorea among patients with Huntington disease: A randomized clinical trial. JAMA 2016; 316: 40-50.
Plante-Bordeneuve V, Said G. Familial amyloid polyneuropathy. Lancet Neurol 2011; 10: 1086-97.
Pinney JH, Whelan CJ, Petrie A, et al. Senile systemic amyloidosis: clinical features at presentation and outcome. J Am Heart Assoc 2013; 2e000098
Paulson HL. The spinocerebellar ataxias. J Neuroophthalmol 2009; 29: 227-37.
Yang S, Li XJ, Li S. Molecular mechanisms underlying Spinocerebellar Ataxia 17 (SCA17) pathogenesis. Rare Dis 2016; 4e1223580
Rhodes LE, Freeman BK, Auh S, et al. Clinical features of spinal and bulbar muscular atrophy. Brain 2009; 132: 3242-51.
Jankovic J, Clarence-Smith K. Tetrabenazine for the treatment of chorea and other hyperkinetic movement disorders. Expert Rev Neurother 2011; 11: 1509-23.
Bonuccelli U, Ceravolo R, Maremmani C, et al. Clozapine in Huntington’s chorea. Neurology 1994; 44: 821-3.
Duff K, Beglinger LJ, O’Rourke ME, et al. Risperidone and the treatment of psychiatric, motor, and cognitive symptoms in Huntington’s disease. Ann Clin Psychiatry 2008; 20: 1-3.
Bonelli RM, Niederwieser G, Tribl GG, et al. High-dose olanzapine in Huntington’s disease. Int Clin Psychopharmacol 2002; 17: 91-3.
Alpay M, Koroshetz WJ. Quetiapine in the treatment of behavioral disturbances in patients with Huntington’s disease. Psychosomatics 2006; 47: 70-2.
Brusa L, Orlacchio A, Moschella V, et al. Treatment of the symptoms of Huntington’s disease: preliminary results comparing aripiprazole and tetrabenazine. Mov Disord 2009; 24: 126-9.
Beister A, Kraus P, Kuhn W, et al. The N-methyl-D-aspartate antagonist memantine retards progression of Huntington’s disease. J Neural Transm Suppl 2004; 117-22.
Verhagen Metman L, Morris MJ, Farmer C, et al. Huntington’s disease: a randomized, controlled trial using the NMDA-antagonist amantadine. Neurology 2002; 59: 694-9.
Landwehrmeyer GB, Dubois B, de Yebenes JG, et al. Riluzole in Huntington’s disease: a 3-year, randomized controlled study. Ann Neurol 2007; 62: 262-72.
Bonelli RM. Mirtazapine in suicidal Huntington’s disease. Ann Pharmacother 2003; 37: 452.
Patel SV, Tariot PN, Asnis J. L-Deprenyl augmentation of fluoxetine in a patient with Huntington’s disease. Ann Clin Psychiatry 1996; 8: 23-6.
Fujikake N, Nagai Y, Popiel HA, et al. Heat shock transcription factor 1-activating compounds suppress polyglutamine-induced neurodegeneration through induction of multiple molecular chaperones. J Biol Chem 2008; 283: 26188-97.
Labbadia J, Cunliffe H, Weiss A, et al. Altered chromatin architecture underlies progressive impairment of the heat shock response in mouse models of Huntington disease. J Clin Invest 2011; 121: 3306-19.
Maheshwari M, Bhutani S, Das A, et al. Dexamethasone induces heat shock response and slows down disease progression in mouse and fly models of Huntington’s disease. Hum Mol Genet 2014; 23: 2737-51.
Machida Y, Okada T, Kurosawa M, et al. rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse. Biochem Biophys Res Commun 2006; 343: 190-7.
Franich NR, Fitzsimons HL, Fong DM, et al. AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington’s disease. Mol Ther 2008; 16: 947-56.
Boudreau RL, Martins I, Davidson BL. Artificial microRNAs as siRNA shuttles: Improved safety as compared to shRNAs in vitro and in vivo. Mol Ther 2009; 17: 169-75.
Dufour BD, Smith CA, Clark RL, et al. Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington’s disease mice. Mol Ther 2014; 22: 797-810.
Stanek LM, Yang W, Angus S, et al. Antisense oligonucleotide-mediated correction of transcriptional dysregulation is correlated with behavioral benefits in the YAC128 mouse model of Huntington’s disease. J Huntingtons Dis 2013; 2: 217-28.
Ostergaard ME, Southwell AL, Kordasiewicz H, et al. Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS. Nucleic Acids Res 2013; 41: 9634-50.