{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T14:18:08Z","timestamp":1792160288119,"version":"4.4.0"},"reference-count":198,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2015,6,1]],"date-time":"2015-06-01T00:00:00Z","timestamp":1433116800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2015,3,20]],"date-time":"2015-03-20T00:00:00Z","timestamp":1426809600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"name":"AFMT\u00e9l\u00e9thon","award":["17295"],"award-info":[{"award-number":["17295"]}]},{"DOI":"10.13039\/501100001826","name":"ZonMw","doi-asserted-by":"publisher","award":["40-41900-98-018"],"award-info":[{"award-number":["40-41900-98-018"]}],"id":[{"id":"10.13039\/501100001826","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Hersenstichting\/ Brugling","award":["BG2013-03"],"award-info":[{"award-number":["BG2013-03"]}]},{"name":"AtaxiaUK"},{"name":"pati\u00ebntenvereniging Autosomaal Dominante Cerebellaire Ataxia (ADCA)"},{"name":"European Community\u2019s Seventh Framework Programme","award":["FP7\/2007-2013"],"award-info":[{"award-number":["FP7\/2007-2013"]}]},{"name":"European Community\u2019s Seventh Framework Programme","award":["2012-305121"],"award-info":[{"award-number":["2012-305121"]}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Advanced Drug Delivery Reviews"],"published-print":{"date-parts":[[2015,6]]},"DOI":"10.1016\/j.addr.2015.03.008","type":"journal-article","created":{"date-parts":[[2015,4,17]],"date-time":"2015-04-17T13:59:51Z","timestamp":1429279191000},"page":"90-103","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":270,"special_numbering":"C","title":["Antisense oligonucleotides in therapy for neurodegenerative disorders"],"prefix":"10.1016","volume":"87","author":[{"given":"Melvin M.","family":"Evers","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lodewijk J.A.","family":"Toonen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Willeke M.C.","family":"van Roon-Mom","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.addr.2015.03.008_bb0005","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1073\/pnas.75.1.285","article-title":"Inhibition of Rous sarcoma viral RNA translation by a specific oligodeoxyribonucleotide","volume":"75","author":"Stephenson","year":"1978","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.addr.2015.03.008_bb0010","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1089\/nat.2013.0454","article-title":"A chemical view of oligonucleotides for exon skipping and related drug applications","volume":"24","author":"Jarver","year":"2014","journal-title":"Nucleic Acid Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0015","doi-asserted-by":"crossref","first-page":"705","DOI":"10.1016\/j.neuroscience.2004.11.038","article-title":"Spinal distribution and metabolism of 2\u2032-O-(2-methoxyethyl)-modified oligonucleotides after intrathecal administration in rats","volume":"131","author":"Butler","year":"2005","journal-title":"Neuroscience"},{"key":"10.1016\/j.addr.2015.03.008_bb0020","doi-asserted-by":"crossref","first-page":"4665","DOI":"10.1073\/pnas.90.10.4665","article-title":"Stability, clearance, and disposition of intraventricularly administered oligodeoxynucleotides: implications for therapeutic application within the central nervous system","volume":"90","author":"Whitesell","year":"1993","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.addr.2015.03.008_bb0025","doi-asserted-by":"crossref","first-page":"3977","DOI":"10.2174\/138161209789649295","article-title":"RNAi applications in therapy development for neurodegenerative disease","volume":"15","author":"Maxwell","year":"2009","journal-title":"Curr. Pharm. Des."},{"key":"10.1016\/j.addr.2015.03.008_bb0030","doi-asserted-by":"crossref","first-page":"554","DOI":"10.1602\/neurorx.2.4.554","article-title":"Drug metabolism and pharmacokinetics, the blood\u2013brain barrier, and central nervous system drug discovery","volume":"2","author":"Alavijeh","year":"2005","journal-title":"NeuroRx"},{"key":"10.1016\/j.addr.2015.03.008_bb0035","first-page":"600","article-title":"Physiology and pharmacological role of the blood\u2013brain barrier","volume":"60","author":"Bernacki","year":"2008","journal-title":"Pharmacol. Rep."},{"key":"10.1016\/j.addr.2015.03.008_bb0040","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.nbd.2009.09.023","article-title":"The blood\u2013brain barrier","volume":"37","author":"Palmer","year":"2010","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.addr.2015.03.008_bb0045","doi-asserted-by":"crossref","first-page":"194","DOI":"10.1002\/ana.22421","article-title":"The blood\u2013spinal cord barrier: morphology and clinical implications","volume":"70","author":"Bartanusz","year":"2011","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.addr.2015.03.008_bb0050","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1007\/s00281-009-0177-0","article-title":"The blood\u2013brain and the blood\u2013cerebrospinal fluid barriers: function and dysfunction","volume":"31","author":"Engelhardt","year":"2009","journal-title":"Semin. Immunopathol."},{"key":"10.1016\/j.addr.2015.03.008_bb0055","doi-asserted-by":"crossref","first-page":"209","DOI":"10.1016\/j.jneuroim.2006.05.012","article-title":"Isolation and culture of microvascular endothelial cells from murine spinal cord","volume":"177","author":"Ge","year":"2006","journal-title":"J. Neuroimmunol."},{"key":"10.1016\/j.addr.2015.03.008_bb0060","doi-asserted-by":"crossref","first-page":"367","DOI":"10.1016\/j.nbd.2006.10.006","article-title":"Blood\u2013brain barrier disruption results in delayed functional and structural alterations in the rat neocortex","volume":"25","author":"Tomkins","year":"2007","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.addr.2015.03.008_bb0065","doi-asserted-by":"crossref","first-page":"E1035","DOI":"10.1073\/pnas.1401595111","article-title":"Blood\u2013spinal cord barrier disruption contributes to early motor\u2013neuron degeneration in ALS-model mice","volume":"111","author":"Winkler","year":"2014","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.addr.2015.03.008_bb0070","doi-asserted-by":"crossref","first-page":"2638","DOI":"10.1021\/ci0600814","article-title":"In silico prediction of blood\u2013brain barrier permeation using the calculated molecular cross-sectional area as main parameter","volume":"46","author":"Gerebtzoff","year":"2006","journal-title":"J. Chem. Inf. Model."},{"key":"10.1016\/j.addr.2015.03.008_bb0075","doi-asserted-by":"crossref","first-page":"157","DOI":"10.3109\/10611860903548354","article-title":"Biopharmaceutical drug targeting to the brain","volume":"18","author":"Pardridge","year":"2010","journal-title":"J. Drug Target."},{"key":"10.1016\/j.addr.2015.03.008_bb0080","doi-asserted-by":"crossref","first-page":"7595","DOI":"10.1073\/pnas.88.17.7595","article-title":"Pharmacokinetics, biodistribution, and stability of oligodeoxynucleotide phosphorothioates in mice","volume":"88","author":"Agrawal","year":"1991","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.addr.2015.03.008_bb0085","doi-asserted-by":"crossref","first-page":"1113","DOI":"10.1016\/S0022-3565(24)29640-3","article-title":"Delivery across the blood\u2013brain barrier of antisense directed against amyloid beta: reversal of learning and memory deficits in mice overexpressing amyloid precursor protein","volume":"297","author":"Banks","year":"2001","journal-title":"J. Pharmacol. Exp. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0090","doi-asserted-by":"crossref","first-page":"1181","DOI":"10.1016\/S0022-3565(25)39459-0","article-title":"Disposition of the 14C-labeled phosphorothioate oligonucleotide ISIS 2105 after intravenous administration to rats","volume":"267","author":"Cossum","year":"1993","journal-title":"J. Pharmacol. Exp. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0095","doi-asserted-by":"crossref","first-page":"1005","DOI":"10.3233\/JAD-131883","article-title":"Central and peripheral administration of antisense oligonucleotide targeting amyloid-beta protein precursor improves learning and memory and reduces neuroinflammatory cytokines in Tg2576 (AbetaPPswe) mice","volume":"40","author":"Farr","year":"2014","journal-title":"J. Alzheimers Dis."},{"key":"10.1016\/j.addr.2015.03.008_bb0100","doi-asserted-by":"crossref","first-page":"54","DOI":"10.1016\/j.drudis.2006.10.013","article-title":"Blood\u2013brain barrier delivery","volume":"12","author":"Pardridge","year":"2007","journal-title":"Drug Discov. Today"},{"key":"10.1016\/j.addr.2015.03.008_bb0105","first-page":"948","article-title":"Imaging gene expression in the brain in vivo in a transgenic mouse model of Huntington's disease with an antisense radiopharmaceutical and drug-targeting technology","volume":"43","author":"Lee","year":"2002","journal-title":"J. Nucl. Med."},{"key":"10.1016\/j.addr.2015.03.008_bb0110","first-page":"340","article-title":"An aquaporin 4 antisense oligonucleotide loaded, brain targeted nanoparticulate system design","volume":"69","author":"Kozlu","year":"2014","journal-title":"Pharmazie"},{"key":"10.1016\/j.addr.2015.03.008_bb0115","doi-asserted-by":"crossref","first-page":"10444","DOI":"10.1016\/S0021-9258(17)34080-2","article-title":"The third helix of the Antennapedia homeodomain translocates through biological membranes","volume":"269","author":"Derossi","year":"1994","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0120","doi-asserted-by":"crossref","first-page":"220","DOI":"10.2174\/1389201015666140617094952","article-title":"Harnessing the capacity of cell-penetrating peptides for drug delivery to the central nervous system","volume":"15","author":"Kang","year":"2014","journal-title":"Curr. Pharm. Biotechnol."},{"key":"10.1016\/j.addr.2015.03.008_bb0125","doi-asserted-by":"crossref","first-page":"3597","DOI":"10.2174\/138161205774580796","article-title":"Cell-penetrating peptides: mechanisms and applications","volume":"11","author":"El-Andaloussi","year":"2005","journal-title":"Curr. Pharm. Des."},{"key":"10.1016\/j.addr.2015.03.008_bb0130","doi-asserted-by":"crossref","first-page":"3151","DOI":"10.1093\/hmg\/ddr217","article-title":"Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum","volume":"20","author":"Du","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2015.03.008_bb0135","doi-asserted-by":"crossref","first-page":"1325","DOI":"10.1021\/bc070060v","article-title":"Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide\u2013morpholino oligomer conjugate","volume":"18","author":"Amantana","year":"2007","journal-title":"Bioconjug. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0140","doi-asserted-by":"crossref","first-page":"2296","DOI":"10.1016\/j.bbamem.2010.02.012","article-title":"Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy","volume":"1798","author":"Moulton","year":"2010","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.addr.2015.03.008_bb0145","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1038\/nbt.1807","article-title":"Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes","volume":"29","author":"Alvarez-Erviti","year":"2011","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.addr.2015.03.008_bb0150","doi-asserted-by":"crossref","first-page":"654","DOI":"10.1038\/ncb1596","article-title":"Exosome-mediated transfer of mRNAs and microRNAs is a novel mechanism of genetic exchange between cells","volume":"9","author":"Valadi","year":"2007","journal-title":"Nat. Cell Biol."},{"key":"10.1016\/j.addr.2015.03.008_bb0155","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1038\/nature05901","article-title":"Transvascular delivery of small interfering RNA to the central nervous system","volume":"448","author":"Kumar","year":"2007","journal-title":"Nature"},{"key":"10.1016\/j.addr.2015.03.008_bb0160","doi-asserted-by":"crossref","first-page":"1031","DOI":"10.1016\/j.neuron.2012.05.009","article-title":"Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis","volume":"74","author":"Kordasiewicz","year":"2012","journal-title":"Neuron"},{"key":"10.1016\/j.addr.2015.03.008_bb0165","doi-asserted-by":"crossref","first-page":"142","DOI":"10.1016\/j.clineuro.2014.05.024","article-title":"Intrathecal baclofen therapy for the symptomatic treatment of hereditary spastic paraplegia","volume":"123","author":"Margetis","year":"2014","journal-title":"Clin. Neurol. Neurosurg."},{"key":"10.1016\/j.addr.2015.03.008_bb0170","doi-asserted-by":"crossref","first-page":"461","DOI":"10.1111\/papr.12111","article-title":"Intrathecal drug administration in chronic pain syndromes","volume":"14","author":"Ver Donck","year":"2014","journal-title":"Pain Pract."},{"key":"10.1016\/j.addr.2015.03.008_bb0175","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1016\/S1474-4422(13)70061-9","article-title":"An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study","volume":"12","author":"Miller","year":"2013","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.addr.2015.03.008_bb0180","series-title":"An Open-label Safety, Tolerability, and Dose-range Finding Study of ISIS SMNRx in Patients With Spinal Muscular Atrophy, ClinicalTrials. gov NCT01494701","author":"Isis Pharmaceuticals","year":"2011"},{"key":"10.1016\/j.addr.2015.03.008_bb0185","doi-asserted-by":"crossref","first-page":"1243","DOI":"10.1126\/science.1136281","article-title":"Human neuroblasts migrate to the olfactory bulb via a lateral ventricular extension","volume":"315","author":"Curtis","year":"2007","journal-title":"Science"},{"key":"10.1016\/j.addr.2015.03.008_bb0190","doi-asserted-by":"crossref","first-page":"789","DOI":"10.3233\/JAD-122308","article-title":"Sex and ApoE genotype differences in treatment response to two doses of intranasal insulin in adults with mild cognitive impairment or Alzheimer's disease","volume":"35","author":"Claxton","year":"2013","journal-title":"J. Alzheimers Dis."},{"key":"10.1016\/j.addr.2015.03.008_bb0195","doi-asserted-by":"crossref","first-page":"9220","DOI":"10.1016\/j.biomaterials.2013.08.036","article-title":"Delivery of siRNA to the brain using a combination of nose-to-brain delivery and cell-penetrating peptide-modified nano-micelles","volume":"34","author":"Kanazawa","year":"2013","journal-title":"Biomaterials"},{"key":"10.1016\/j.addr.2015.03.008_bb0200","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1215\/15228517-2007-052","article-title":"New therapeutic approach for brain tumors: intranasal delivery of telomerase inhibitor GRN163","volume":"10","author":"Hashizume","year":"2008","journal-title":"Neuro Oncol."},{"key":"10.1016\/j.addr.2015.03.008_bb0205","first-page":"347","article-title":"Antisense oligonucleotides: basic concepts and mechanisms","volume":"1","author":"Dias","year":"2002","journal-title":"Mol. Cancer Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0210","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1089\/ard.1991.1.141","article-title":"Substrate specificity and kinetics of degradation of antisense oligonucleotides by a 3\u2032 exonuclease in plasma","volume":"1","author":"Eder","year":"1991","journal-title":"Antisense Res. Dev."},{"key":"10.1016\/j.addr.2015.03.008_bb0215","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1089\/ard.1991.1.11","article-title":"Pathways of degradation and mechanism of action of antisense oligonucleotides in Xenopus laevis embryos","volume":"1","author":"Dagle","year":"1991","journal-title":"Antisense Res. Dev."},{"key":"10.1016\/j.addr.2015.03.008_bb0220","doi-asserted-by":"crossref","first-page":"1137","DOI":"10.1126\/science.165.3898.1137","article-title":"Interferon induction increased through chemical modification of a synthetic polyribonucleotide","volume":"165","author":"De Clercq","year":"1969","journal-title":"Science"},{"key":"10.1016\/j.addr.2015.03.008_bb0225","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1016\/0165-022X(90)90084-P","article-title":"Oligodeoxynucleoside phosphorothioate stability in subcellular extracts, culture media, sera and cerebrospinal fluid","volume":"20","author":"Campbell","year":"1990","journal-title":"J. Biochem. Biophys. Methods"},{"key":"10.1016\/j.addr.2015.03.008_bb0230","doi-asserted-by":"crossref","first-page":"211","DOI":"10.1089\/ard.1992.2.211","article-title":"Cellular uptake and subcellular distribution of phosphorothioate oligonucleotides into cultured cells","volume":"2","author":"Iversen","year":"1992","journal-title":"Antisense Res. Dev."},{"key":"10.1016\/j.addr.2015.03.008_bb0235","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1016\/0167-0115(95)00096-T","article-title":"Cellular uptake of intracerebrally administered oligodeoxynucleotides in mouse brain","volume":"59","author":"Ogawa","year":"1995","journal-title":"Regul. Pept."},{"key":"10.1016\/j.addr.2015.03.008_bb0240","doi-asserted-by":"crossref","first-page":"17181","DOI":"10.1074\/jbc.M311683200","article-title":"Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs","volume":"279","author":"Wu","year":"2004","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0245","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1146\/annurev.pharmtox.010909.105654","article-title":"RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform","volume":"50","author":"Bennett","year":"2010","journal-title":"Annu. Rev. Pharmacol. Toxicol."},{"key":"10.1016\/j.addr.2015.03.008_bb0250","doi-asserted-by":"crossref","first-page":"1494","DOI":"10.1111\/j.1742-4658.2009.06908.x","article-title":"Ribonuclease H: the enzymes in eukaryotes","volume":"276","author":"Cerritelli","year":"2009","journal-title":"FEBS J."},{"key":"10.1016\/j.addr.2015.03.008_bb0255","doi-asserted-by":"crossref","first-page":"829","DOI":"10.1093\/nar\/24.5.829","article-title":"NMR investigations of duplex stability of phosphorothioate and phosphorodithioate DNA analogues modified in both strands","volume":"24","author":"Jaroszewski","year":"1996","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0260","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1016\/S0167-4781(99)00140-2","article-title":"A review of the issues in the pharmacokinetics and toxicology of phosphorothioate antisense oligonucleotides","volume":"1489","author":"Levin","year":"1999","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.addr.2015.03.008_bb0265","doi-asserted-by":"crossref","first-page":"26801","DOI":"10.1016\/S0021-9258(18)47090-1","article-title":"Effect of phosphorothioate modification of oligodeoxynucleotides on specific protein binding","volume":"269","author":"Brown","year":"1994","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0270","doi-asserted-by":"crossref","first-page":"2620","DOI":"10.1074\/jbc.270.6.2620","article-title":"Phosphorothioate oligodeoxynucleotides bind to basic fibroblast growth factor, inhibit its binding to cell surface receptors, and remove it from low affinity binding sites on extracellular matrix","volume":"270","author":"Guvakova","year":"1995","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0275","doi-asserted-by":"crossref","first-page":"201","DOI":"10.1089\/ard.1994.4.201","article-title":"Complement activation and hemodynamic changes following intravenous administration of phosphorothioate oligonucleotides in the monkey","volume":"4","author":"Galbraith","year":"1994","journal-title":"Antisense Res. Dev."},{"key":"10.1016\/j.addr.2015.03.008_bb0280","first-page":"717","article-title":"Clearance kinetics, biodistribution, and organ saturability of phosphorothioate oligodeoxynucleotides in mice","volume":"149","author":"Rifai","year":"1996","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.addr.2015.03.008_bb0285","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1089\/oli.2006.16.169","article-title":"Plasma protein binding of an antisense oligonucleotide targeting human ICAM-1 (ISIS 2302)","volume":"16","author":"Watanabe","year":"2006","journal-title":"Oligonucleotides"},{"key":"10.1016\/j.addr.2015.03.008_bb0290","doi-asserted-by":"crossref","first-page":"657","DOI":"10.1016\/S0006-2952(97)00190-1","article-title":"Pharmacokinetics, metabolism, and elimination of a 20-mer phosphorothioate oligodeoxynucleotide (CGP 69846A) after intravenous and subcutaneous administration","volume":"54","author":"Phillips","year":"1997","journal-title":"Biochem. Pharmacol."},{"key":"10.1016\/j.addr.2015.03.008_bb0295","doi-asserted-by":"crossref","first-page":"4429","DOI":"10.1093\/nar\/25.22.4429","article-title":"The ups and downs of nucleic acid duplex stability: structure\u2013stability studies on chemically-modified DNA:RNA duplexes","volume":"25","author":"Freier","year":"1997","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0300","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1016\/1074-5521(94)90039-6","article-title":"Stabilizing effects of the RNA 2\u2032-substituent: crystal structure of an oligodeoxynucleotide duplex containing 2\u2032-O-methylated adenosines","volume":"1","author":"Lubini","year":"1994","journal-title":"Chem. Biol."},{"key":"10.1016\/j.addr.2015.03.008_bb0305","doi-asserted-by":"crossref","first-page":"1715","DOI":"10.1074\/jbc.274.3.1715","article-title":"Characterization of a potent and specific class of antisense oligonucleotide inhibitor of human protein kinase C-alpha expression","volume":"274","author":"McKay","year":"1999","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0310","doi-asserted-by":"crossref","first-page":"890","DOI":"10.1016\/S0022-3565(24)38831-7","article-title":"Pharmacokinetic properties of 2\u2032-O-(2-methoxyethyl)-modified oligonucleotide analogs in rats","volume":"296","author":"Geary","year":"2001","journal-title":"J. Pharmacol. Exp. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0315","doi-asserted-by":"crossref","first-page":"173","DOI":"10.1016\/0006-2952(95)02177-9","article-title":"Effect of different chemically modified oligodeoxynucleotides on immune stimulation","volume":"51","author":"Zhao","year":"1996","journal-title":"Biochem. Pharmacol."},{"key":"10.1016\/j.addr.2015.03.008_bb0320","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/S0169-328X(98)00185-5","article-title":"Modification of phosphorothioate oligonucleotides yields potent analogs with minimal toxicity for antisense experiments in the CNS","volume":"62","author":"Peng","year":"1998","journal-title":"Brain Res. Mol. Brain Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0325","doi-asserted-by":"crossref","first-page":"14514","DOI":"10.1016\/S0021-9258(19)85268-7","article-title":"Evaluation of 2\u2032-modified oligonucleotides containing 2\u2032-deoxy gaps as antisense inhibitors of gene expression","volume":"268","author":"Monia","year":"1993","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0330","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1016\/S0022-3956(98)80047-2","article-title":"Differential behavioural effects of chronic infusion of CRH 1 and CRH 2 receptor antisense oligonucleotides into the rat brain","volume":"33","author":"Liebsch","year":"1999","journal-title":"J. Psychiatr. Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0335","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1016\/S0165-3806(00)00004-3","article-title":"Antisense knockdown of calcium-dependent K+ channels in developing cerebellar Purkinje neurons","volume":"120","author":"Muller","year":"2000","journal-title":"Brain Res. Dev. Brain Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0340","doi-asserted-by":"crossref","first-page":"559","DOI":"10.1016\/j.imbio.2009.09.003","article-title":"Alternating 2\u2032-O-ribose methylation is a universal approach for generating non-stimulatory siRNA by acting as TLR7 antagonist","volume":"215","author":"Hamm","year":"2010","journal-title":"Immunobiology"},{"key":"10.1016\/j.addr.2015.03.008_bb0345","doi-asserted-by":"crossref","first-page":"2079","DOI":"10.1523\/JNEUROSCI.5348-10.2011","article-title":"Region-specific involvement of BDNF secretion and synthesis in conditioned taste aversion memory formation","volume":"31","author":"Ma","year":"2011","journal-title":"J. Neurosci."},{"key":"10.1016\/j.addr.2015.03.008_bb0350","doi-asserted-by":"crossref","first-page":"1663","DOI":"10.1038\/sj.mt.6300240","article-title":"2\u2032-O-methyl-modified RNAs act as TLR7 antagonists","volume":"15","author":"Robbins","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0355","doi-asserted-by":"crossref","first-page":"1634","DOI":"10.1101\/gad.1941310","article-title":"Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model","volume":"24","author":"Hua","year":"2010","journal-title":"Genes Dev."},{"key":"10.1016\/j.addr.2015.03.008_bb0360","doi-asserted-by":"crossref","first-page":"1041","DOI":"10.1081\/NCN-120022731","article-title":"Nuclease stability of LNA oligonucleotides and LNA\u2013DNA chimeras","volume":"22","author":"Frieden","year":"2003","journal-title":"Nucleosides Nucleotides Nucleic Acids"},{"key":"10.1016\/j.addr.2015.03.008_bb0365","doi-asserted-by":"crossref","first-page":"5160","DOI":"10.1093\/nar\/gkf651","article-title":"Antisense inhibition of gene expression in cells by oligonucleotides incorporating locked nucleic acids: effect of mRNA target sequence and chimera design","volume":"30","author":"Braasch","year":"2002","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0370","doi-asserted-by":"crossref","first-page":"9973","DOI":"10.1021\/bi025907j","article-title":"Implications of high-affinity hybridization by locked nucleic acid oligomers for inhibition of human telomerase","volume":"41","author":"Elayadi","year":"2002","journal-title":"Biochemistry"},{"key":"10.1016\/j.addr.2015.03.008_bb0375","doi-asserted-by":"crossref","first-page":"1911","DOI":"10.1093\/nar\/30.9.1911","article-title":"Design of antisense oligonucleotides stabilized by locked nucleic acids","volume":"30","author":"Kurreck","year":"2002","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0380","doi-asserted-by":"crossref","first-page":"2164","DOI":"10.1021\/ja0168763","article-title":"Alpha-L-ribo-configured locked nucleic acid (alpha-L-LNA): synthesis and properties","volume":"124","author":"Sorensen","year":"2002","journal-title":"J. Am. Chem. Soc."},{"key":"10.1016\/j.addr.2015.03.008_bb0385","doi-asserted-by":"crossref","first-page":"687","DOI":"10.1093\/nar\/gkl1071","article-title":"Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals","volume":"35","author":"Swayze","year":"2007","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0390","doi-asserted-by":"crossref","first-page":"5633","DOI":"10.1073\/pnas.97.10.5633","article-title":"Potent and nontoxic antisense oligonucleotides containing locked nucleic acids","volume":"97","author":"Wahlestedt","year":"2000","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.addr.2015.03.008_bb0395","doi-asserted-by":"crossref","first-page":"30613","DOI":"10.1074\/jbc.M603346200","article-title":"Antisense inhibition of RNase P: mechanistic aspects and application to live bacteria","volume":"281","author":"Gruegelsiepe","year":"2006","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0400","doi-asserted-by":"crossref","first-page":"13500","DOI":"10.1021\/ja804727a","article-title":"Oligonucleotide\u2013oligospermine conjugates (zip nucleic acids): a convenient means of finely tuning hybridization temperatures","volume":"130","author":"Noir","year":"2008","journal-title":"J. Am. Chem. Soc."},{"key":"10.1016\/j.addr.2015.03.008_bb0405","doi-asserted-by":"crossref","first-page":"8404","DOI":"10.1021\/ja200312y","article-title":"Antisense and antigene inhibition of gene expression by cell-permeable oligonucleotide\u2013oligospermine conjugates","volume":"133","author":"Gagnon","year":"2011","journal-title":"J. Am. Chem. Soc."},{"key":"10.1016\/j.addr.2015.03.008_bb0410","doi-asserted-by":"crossref","first-page":"5993","DOI":"10.1021\/ja025569+","article-title":"Watson\u2013Crick base-pairing properties of tricyclo-DNA","volume":"124","author":"Renneberg","year":"2002","journal-title":"J. Am. Chem. Soc."},{"key":"10.1016\/j.addr.2015.03.008_bb0415","doi-asserted-by":"crossref","first-page":"2751","DOI":"10.1093\/nar\/gkf412","article-title":"Antisense properties of tricyclo-DNA","volume":"30","author":"Renneberg","year":"2002","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0420","doi-asserted-by":"crossref","first-page":"270","DOI":"10.1038\/nm.3765","article-title":"Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers","volume":"21","author":"Goyenvalle","year":"2015","journal-title":"Nat. Med."},{"key":"10.1016\/j.addr.2015.03.008_bb0425","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1021\/jm00059a007","article-title":"Uniformly modified 2\u2032-deoxy-2\u2032-fluoro phosphorothioate oligonucleotides as nuclease-resistant antisense compounds with high affinity and specificity for RNA targets","volume":"36","author":"Kawasaki","year":"1993","journal-title":"J. Med. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0430","doi-asserted-by":"crossref","first-page":"553","DOI":"10.1093\/nass\/nrn280","article-title":"Design, synthesis and evaluation of constrained methoxyethyl (cMOE) and constrained ethyl (cEt) nucleoside analogs","author":"Seth","year":"2008","journal-title":"Nucleic Acids Symp. Ser. (Oxf.)"},{"key":"10.1016\/j.addr.2015.03.008_bb0435","doi-asserted-by":"crossref","first-page":"2093","DOI":"10.1038\/mt.2014.153","article-title":"In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides","volume":"22","author":"Southwell","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0440","doi-asserted-by":"crossref","first-page":"1497","DOI":"10.1126\/science.1962210","article-title":"Sequence-selective recognition of DNA by strand displacement with a thymine-substituted polyamide","volume":"254","author":"Nielsen","year":"1991","journal-title":"Science"},{"key":"10.1016\/j.addr.2015.03.008_bb0445","doi-asserted-by":"crossref","first-page":"566","DOI":"10.1038\/365566a0","article-title":"PNA hybridizes to complementary oligonucleotides obeying the Watson\u2013Crick hydrogen-bonding rules","volume":"365","author":"Egholm","year":"1993","journal-title":"Nature"},{"key":"10.1016\/j.addr.2015.03.008_bb0450","doi-asserted-by":"crossref","first-page":"1310","DOI":"10.1016\/0006-2952(94)90171-6","article-title":"Stability of peptide nucleic acids in human serum and cellular extracts","volume":"48","author":"Demidov","year":"1994","journal-title":"Biochem. Pharmacol."},{"key":"10.1016\/j.addr.2015.03.008_bb0455","doi-asserted-by":"crossref","first-page":"4792","DOI":"10.1093\/nar\/27.24.4792","article-title":"Thermodynamic comparison of PNA\/DNA and DNA\/DNA hybridization reactions at ambient temperature","volume":"27","author":"Schwarz","year":"1999","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0460","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1016\/0014-5793(95)00409-3","article-title":"Phospholipid membrane permeability of peptide nucleic acid","volume":"375","author":"Wittung","year":"1995","journal-title":"FEBS Lett."},{"key":"10.1016\/j.addr.2015.03.008_bb0465","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1016\/0968-0896(95)00171-9","article-title":"Peptide nucleic acids (PNA): synthesis, properties and potential applications","volume":"4","author":"Hyrup","year":"1996","journal-title":"Bioorg. Med. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0470","doi-asserted-by":"crossref","first-page":"4910","DOI":"10.1093\/nar\/26.21.4910","article-title":"A peptide nucleic acid (PNA) is more rapidly internalized in cultured neurons when coupled to a retro-inverso delivery peptide. The antisense activity depresses the target mRNA and protein in magnocellular oxytocin neurons","volume":"26","author":"Aldrian-Herrada","year":"1998","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0475","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1002\/psc.1198","article-title":"Cellular uptake and biological activity of peptide nucleic acids conjugated with peptides with and without cell-penetrating ability","volume":"16","author":"Turner","year":"2010","journal-title":"J. Pept. Sci."},{"key":"10.1016\/j.addr.2015.03.008_bb0480","doi-asserted-by":"crossref","first-page":"280","DOI":"10.1016\/S0014-5793(97)01575-5","article-title":"Specific gene blockade shows that peptide nucleic acids readily enter neuronal cells in vivo","volume":"421","author":"Tyler","year":"1998","journal-title":"FEBS Lett."},{"key":"10.1016\/j.addr.2015.03.008_bb0485","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1089\/108729002760070803","article-title":"Pharmacokinetics and tissue distribution of a peptide nucleic acid after intravenous administration","volume":"12","author":"McMahon","year":"2002","journal-title":"Antisense Nucleic Acid Drug Dev."},{"key":"10.1016\/j.addr.2015.03.008_bb0490","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1089\/oli.1.1997.7.187","article-title":"Morpholino antisense oligomers: design, preparation, and properties","volume":"7","author":"Summerton","year":"1997","journal-title":"Antisense Nucleic Acid Drug Dev."},{"key":"10.1016\/j.addr.2015.03.008_bb0495","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1089\/oli.1.1996.6.267","article-title":"Resistance of morpholino phosphorodiamidate oligomers to enzymatic degradation","volume":"6","author":"Hudziak","year":"1996","journal-title":"Antisense Nucleic Acid Drug Dev."},{"key":"10.1016\/j.addr.2015.03.008_bb0500","doi-asserted-by":"crossref","first-page":"3930","DOI":"10.1158\/1078-0432.CCR-04-2091","article-title":"In vivo bioavailability and pharmacokinetics of a c-MYC antisense phosphorodiamidate morpholino oligomer, AVI-4126, in solid tumors","volume":"11","author":"Devi","year":"2005","journal-title":"Clin. Cancer Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0505","doi-asserted-by":"crossref","first-page":"14814","DOI":"10.1073\/pnas.0805676105","article-title":"Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer","volume":"105","author":"Wu","year":"2008","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.addr.2015.03.008_bb0510","doi-asserted-by":"crossref","first-page":"1625","DOI":"10.1093\/hmg\/ddr600","article-title":"A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse","volume":"21","author":"Porensky","year":"2012","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2015.03.008_bb0515","doi-asserted-by":"crossref","first-page":"340","DOI":"10.1016\/j.clinthera.2014.02.004","article-title":"Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy Delta7 mouse model phenotype","volume":"36","author":"Nizzardo","year":"2014","journal-title":"Clin. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0520","doi-asserted-by":"crossref","first-page":"613","DOI":"10.2144\/000113005","article-title":"Vivo-Morpholinos: a non-peptide transporter delivers morpholinos into a wide array of mouse tissues","volume":"45","author":"Morcos","year":"2008","journal-title":"Biotechniques"},{"key":"10.1016\/j.addr.2015.03.008_bb0525","doi-asserted-by":"crossref","first-page":"251","DOI":"10.2144\/000114167","article-title":"Lessons learned from vivo-morpholinos: how to avoid vivo-morpholino toxicity","volume":"56","author":"Ferguson","year":"2014","journal-title":"Biotechniques"},{"key":"10.1016\/j.addr.2015.03.008_bb0530","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1089\/hum.2012.211","article-title":"A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice","volume":"24","author":"Zhou","year":"2013","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0535","doi-asserted-by":"crossref","first-page":"e61472","DOI":"10.1371\/journal.pone.0061472","article-title":"Vivo-morpholinos induced transient knockdown of physical activity related proteins","volume":"8","author":"Ferguson","year":"2013","journal-title":"PLoS ONE"},{"key":"10.1016\/j.addr.2015.03.008_bb0540","doi-asserted-by":"crossref","first-page":"354","DOI":"10.1016\/j.jneumeth.2011.10.009","article-title":"Use of vivo-morpholinos for control of protein expression in the adult rat brain","volume":"203","author":"Reissner","year":"2012","journal-title":"J. Neurosci. Methods"},{"key":"10.1016\/j.addr.2015.03.008_bb0545","doi-asserted-by":"crossref","first-page":"14240","DOI":"10.1073\/pnas.96.25.14240","article-title":"Structural origins of the exonuclease resistance of a zwitterionic RNA","volume":"96","author":"Teplova","year":"1999","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.addr.2015.03.008_bb0550","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1016\/j.neuroscience.2007.01.008","article-title":"Antisense inhibition at the beta-secretase-site of beta-amyloid precursor protein reduces cerebral amyloid and acetyl cholinesterase activity in Tg2576","volume":"146","author":"Chauhan","year":"2007","journal-title":"Neuroscience"},{"key":"10.1016\/j.addr.2015.03.008_bb0555","doi-asserted-by":"crossref","first-page":"e24308","DOI":"10.1371\/journal.pone.0024308","article-title":"Targeting several CAG expansion diseases by a single antisense oligonucleotide","volume":"6","author":"Evers","year":"2011","journal-title":"PLoS ONE"},{"key":"10.1016\/j.addr.2015.03.008_bb0560","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1038\/nbt.1539","article-title":"Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs","volume":"27","author":"Hu","year":"2009","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.addr.2015.03.008_bb0565","doi-asserted-by":"crossref","first-page":"6302","DOI":"10.1093\/hmg\/ddu349","article-title":"Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity","volume":"23","author":"Sun","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2015.03.008_bb0570","doi-asserted-by":"crossref","first-page":"7195","DOI":"10.1073\/pnas.1231012100","article-title":"Allele-specific silencing of dominant disease genes","volume":"100","author":"Miller","year":"2003","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.addr.2015.03.008_bb0575","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1038\/nrd3625","article-title":"RNA therapeutics: beyond RNA interference and antisense oligonucleotides","volume":"11","author":"Kole","year":"2012","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.addr.2015.03.008_bb0580","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1016\/j.nbt.2013.01.005","article-title":"Advances in therapeutic RNA-targeting","volume":"30","author":"van Ommen","year":"2013","journal-title":"N. Biotechnol."},{"key":"10.1016\/j.addr.2015.03.008_bb0585","doi-asserted-by":"crossref","first-page":"860","DOI":"10.1038\/35057062","article-title":"Initial sequencing and analysis of the human genome","volume":"409","author":"Lander","year":"2001","journal-title":"Nature"},{"key":"10.1016\/j.addr.2015.03.008_bb0590","doi-asserted-by":"crossref","first-page":"R74","DOI":"10.1186\/gb-2004-5-10-r74","article-title":"Variation in alternative splicing across human tissues","volume":"5","author":"Yeo","year":"2004","journal-title":"Genome Biol."},{"key":"10.1016\/j.addr.2015.03.008_bb0595","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1186\/1471-2202-14-5","article-title":"Antisense-mediated isoform switching of steroid receptor coactivator-1 in the central nucleus of the amygdala of the mouse brain","volume":"14","author":"Zalachoras","year":"2013","journal-title":"BMC Neurosci."},{"key":"10.1016\/j.addr.2015.03.008_bb0600","doi-asserted-by":"crossref","first-page":"1333","DOI":"10.1128\/MCB.26.4.1333-1346.2006","article-title":"Splicing of a critical exon of human survival motor neuron is regulated by a unique silencer element located in the last intron","volume":"26","author":"Singh","year":"2006","journal-title":"Mol. Cell. Biol."},{"key":"10.1016\/j.addr.2015.03.008_bb0605","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1016\/j.nbd.2013.04.019","article-title":"Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: removal of the CAG containing exon","volume":"58","author":"Evers","year":"2013","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.addr.2015.03.008_bb0610","doi-asserted-by":"crossref","first-page":"6007","DOI":"10.1073\/pnas.0608616104","article-title":"Correction of prototypic ATM splicing mutations and aberrant ATM function with antisense morpholino oligonucleotides","volume":"104","author":"Du","year":"2007","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.addr.2015.03.008_bb0615","doi-asserted-by":"crossref","first-page":"637","DOI":"10.1002\/ana.23982","article-title":"Eteplirsen for the treatment of Duchenne muscular dystrophy","volume":"74","author":"Mendell","year":"2013","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.addr.2015.03.008_bb0620","doi-asserted-by":"crossref","first-page":"987","DOI":"10.1016\/S1474-4422(14)70195-4","article-title":"Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study","volume":"13","author":"Voit","year":"2014","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.addr.2015.03.008_bb0625","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1038\/nature11362","article-title":"Targeting nuclear RNA for in vivo correction of myotonic dystrophy","volume":"488","author":"Wheeler","year":"2012","journal-title":"Nature"},{"key":"10.1016\/j.addr.2015.03.008_bb0630","doi-asserted-by":"crossref","first-page":"122","DOI":"10.1089\/oli.2007.0034","article-title":"In situ entry of oligonucleotides into brain cells can occur through a nucleic acid channel","volume":"17","author":"Shi","year":"2007","journal-title":"Oligonucleotides"},{"key":"10.1016\/j.addr.2015.03.008_bb0635","doi-asserted-by":"crossref","first-page":"284","DOI":"10.1016\/j.bcp.2009.04.013","article-title":"Effect of dose and plasma concentration on liver uptake and pharmacologic activity of a 2\u2032-methoxyethyl modified chimeric antisense oligonucleotide targeting PTEN","volume":"78","author":"Geary","year":"2009","journal-title":"Biochem. Pharmacol."},{"key":"10.1016\/j.addr.2015.03.008_bb0640","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1021\/bc200377d","article-title":"Cellular uptake and intracellular trafficking of antisense and siRNA oligonucleotides","volume":"23","author":"Juliano","year":"2012","journal-title":"Bioconjug. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0645","doi-asserted-by":"crossref","first-page":"4795","DOI":"10.1093\/nar\/gkr089","article-title":"Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes","volume":"39","author":"Koller","year":"2011","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0650","doi-asserted-by":"crossref","first-page":"1210","DOI":"10.1038\/mt.2010.72","article-title":"Preclinical PK and PD studies on 2\u2032-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model","volume":"18","author":"Heemskerk","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0655","doi-asserted-by":"crossref","first-page":"e148","DOI":"10.1038\/mtna.2014.1","article-title":"The dynamics of compound, transcript, and protein effects after treatment with 2OMePS antisense oligonucleotides in mdx mice","volume":"3","author":"Verhaart","year":"2014","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.addr.2015.03.008_bb0660","doi-asserted-by":"crossref","first-page":"46","DOI":"10.1124\/jpet.113.212407","article-title":"Pharmacology of a central nervous system delivered 2\u2032-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates","volume":"350","author":"Rigo","year":"2014","journal-title":"J. Pharmacol. Exp. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0665","doi-asserted-by":"crossref","first-page":"2290","DOI":"10.1172\/JCI25424","article-title":"Antisense oligonucleotide therapy for neurodegenerative disease","volume":"116","author":"Smith","year":"2006","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.addr.2015.03.008_bb0670","doi-asserted-by":"crossref","first-page":"e180","DOI":"10.1038\/mtna.2014.30","article-title":"Antisense-mediated exon skipping decreases tau protein expression: a potential therapy for tauopathies","volume":"3","author":"Sud","year":"2014","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.addr.2015.03.008_bb0675","doi-asserted-by":"crossref","first-page":"548","DOI":"10.1038\/mt.2008.205","article-title":"Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms","volume":"17","author":"Aartsma-Rus","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0680","doi-asserted-by":"crossref","first-page":"349","DOI":"10.1159\/000105156","article-title":"Current insights into molecular mechanisms of Alzheimer disease and their implications for therapeutic approaches","volume":"4","author":"Van Broeck","year":"2007","journal-title":"Neurodegener. Dis."},{"key":"10.1016\/j.addr.2015.03.008_bb0685","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1038\/nrn2967","article-title":"Amyloid-beta and tau \u2014 a toxic pas de deux in Alzheimer's disease","volume":"12","author":"Ittner","year":"2011","journal-title":"Nat. Rev. Neurosci."},{"key":"10.1016\/j.addr.2015.03.008_bb0690","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1097\/00019052-200008000-00006","article-title":"Recent advances in amyotrophic lateral sclerosis","volume":"13","author":"Al-Chalabi","year":"2000","journal-title":"Curr. Opin. Neurol."},{"key":"10.1016\/j.addr.2015.03.008_bb0695","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1038\/nn.3584","article-title":"State of play in amyotrophic lateral sclerosis genetics","volume":"17","author":"Renton","year":"2014","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.addr.2015.03.008_bb0700","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1016\/j.neuron.2011.09.011","article-title":"Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS","volume":"72","author":"DeJesus-Hernandez","year":"2011","journal-title":"Neuron"},{"key":"10.1016\/j.addr.2015.03.008_bb0705","doi-asserted-by":"crossref","first-page":"597","DOI":"10.1016\/j.neuron.2013.02.009","article-title":"RANTing about C9orf72","volume":"77","author":"Lashley","year":"2013","journal-title":"Neuron"},{"key":"10.1016\/j.addr.2015.03.008_bb0710","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1016\/j.neuron.2013.10.015","article-title":"RNA toxicity from the ALS\/FTD C9ORF72 expansion is mitigated by antisense intervention","volume":"80","author":"Donnelly","year":"2013","journal-title":"Neuron"},{"key":"10.1016\/j.addr.2015.03.008_bb0715","doi-asserted-by":"crossref","first-page":"E4530","DOI":"10.1073\/pnas.1318835110","article-title":"Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration","volume":"110","author":"Lagier-Tourenne","year":"2013","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.addr.2015.03.008_bb0720","doi-asserted-by":"crossref","first-page":"721","DOI":"10.1007\/s12035-014-8724-7","article-title":"Antisense oligonucleotide therapy for the treatment of C9ORF72 ALS\/FTD diseases","volume":"50","author":"Riboldi","year":"2014","journal-title":"Mol. Neurobiol."},{"key":"10.1016\/j.addr.2015.03.008_bb0725","doi-asserted-by":"crossref","first-page":"500","DOI":"10.1172\/JCI45130","article-title":"Oligonucleotide therapeutic approaches for Huntington disease","volume":"121","author":"Sah","year":"2011","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.addr.2015.03.008_bb0730","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1016\/S0361-9230(01)00577-9","article-title":"Polyglutamine expansion neurodegenerative disease","volume":"56","author":"Fischbeck","year":"2001","journal-title":"Brain Res. Bull."},{"key":"10.1016\/j.addr.2015.03.008_bb0735","doi-asserted-by":"crossref","first-page":"537","DOI":"10.1016\/S0092-8674(00)80513-9","article-title":"Formation of neuronal intranuclear inclusions underlies the neurological dysfunction in mice transgenic for the HD mutation","volume":"90","author":"Davies","year":"1997","journal-title":"Cell"},{"key":"10.1016\/j.addr.2015.03.008_bb0740","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/s00401-012-1000-x","article-title":"Brain pathology of spinocerebellar ataxias","volume":"124","author":"Seidel","year":"2012","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.addr.2015.03.008_bb0745","doi-asserted-by":"crossref","first-page":"424","DOI":"10.1002\/ana.410370404","article-title":"The adhesion molecule and cytokine profile of multiple sclerosis lesions","volume":"37","author":"Cannella","year":"1995","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.addr.2015.03.008_bb0750","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1111\/j.1600-065X.2007.00536.x","article-title":"Integrin modulation and signaling in leukocyte adhesion and migration","volume":"218","author":"Rose","year":"2007","journal-title":"Immunol. Rev."},{"key":"10.1016\/j.addr.2015.03.008_bb0755","doi-asserted-by":"crossref","first-page":"1722","DOI":"10.1172\/JCI117519","article-title":"The pathophysiologic role of alpha 4 integrins in vivo","volume":"94","author":"Lobb","year":"1994","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.addr.2015.03.008_bb0760","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1016\/j.jneuroim.2004.10.029","article-title":"Antisense oligonucleotide blockade of alpha 4 integrin prevents and reverses clinical symptoms in murine experimental autoimmune encephalomyelitis","volume":"160","author":"Myers","year":"2005","journal-title":"J. Neuroimmunol."},{"key":"10.1016\/j.addr.2015.03.008_bb0765","doi-asserted-by":"crossref","first-page":"e9","DOI":"10.1038\/mtna.2011.6","article-title":"Intracerebral infusion of antisense oligonucleotides into prion-infected mice","volume":"1","author":"Nazor Friberg","year":"2012","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.addr.2015.03.008_bb0770","doi-asserted-by":"crossref","first-page":"618","DOI":"10.1016\/S1474-4422(12)70063-7","article-title":"Sporadic human prion diseases: molecular insights and diagnosis","volume":"11","author":"Puoti","year":"2012","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.addr.2015.03.008_bb0775","doi-asserted-by":"crossref","first-page":"190","DOI":"10.2119\/2006-00073.Karpuj","article-title":"Phosphorothioate oligonucleotides reduce PrP levels and prion infectivity in cultured cells","volume":"13","author":"Karpuj","year":"2007","journal-title":"Mol. Med."},{"key":"10.1016\/j.addr.2015.03.008_bb0780","doi-asserted-by":"crossref","first-page":"1034","DOI":"10.1128\/AAC.50.3.1034-1044.2006","article-title":"Potent antiscrapie activities of degenerate phosphorothioate oligonucleotides","volume":"50","author":"Kocisko","year":"2006","journal-title":"Antimicrob. Agents Chemother."},{"key":"10.1016\/j.addr.2015.03.008_bb0785","doi-asserted-by":"crossref","first-page":"e107434","DOI":"10.1371\/journal.pone.0107434","article-title":"Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients","volume":"9","author":"Skotte","year":"2014","journal-title":"PLoS ONE"},{"key":"10.1016\/j.addr.2015.03.008_bb0790","doi-asserted-by":"crossref","first-page":"2178","DOI":"10.1038\/mt.2011.201","article-title":"Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene\/allele-specific silencing of mutant huntingtin","volume":"19","author":"Carroll","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0795","doi-asserted-by":"crossref","first-page":"9634","DOI":"10.1093\/nar\/gkt725","article-title":"Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS","volume":"41","author":"Ostergaard","year":"2013","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0800","doi-asserted-by":"crossref","first-page":"565","DOI":"10.1146\/annurev.neuro.26.041002.131334","article-title":"Notch and Presenilin: regulated intramembrane proteolysis links development and degeneration","volume":"26","author":"Selkoe","year":"2003","journal-title":"Annu. Rev. Neurosci."},{"key":"10.1016\/j.addr.2015.03.008_bb0805","doi-asserted-by":"crossref","first-page":"R111","DOI":"10.1093\/hmg\/ddq147","article-title":"Spinal muscular atrophy: mechanisms and therapeutic strategies","volume":"19","author":"Lorson","year":"2010","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2015.03.008_bb0810","doi-asserted-by":"crossref","first-page":"72ra18","DOI":"10.1126\/scitranslmed.3001777","article-title":"Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy","volume":"3","author":"Passini","year":"2011","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.addr.2015.03.008_bb0815","doi-asserted-by":"crossref","first-page":"45476","DOI":"10.1074\/jbc.M107632200","article-title":"Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3\u2032 splice site pairing","volume":"276","author":"Lim","year":"2001","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0820","doi-asserted-by":"crossref","first-page":"7633","DOI":"10.1523\/JNEUROSCI.0950-09.2009","article-title":"Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy","volume":"29","author":"Williams","year":"2009","journal-title":"J. Neurosci."},{"key":"10.1016\/j.addr.2015.03.008_bb0825","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1083\/jcb.201207087","article-title":"Antisense-based therapy for the treatment of spinal muscular atrophy","volume":"199","author":"Rigo","year":"2012","journal-title":"J. Cell Biol."},{"key":"10.1016\/j.addr.2015.03.008_bb0830","series-title":"An Open-label Safety, Tolerability and Dose-range Finding Study of Multiple Doses of ISIS SMNRx in Patient With Spinal Muscular Atrophy (SMNRx \u2014 CS2), ClinicalTrials. gov NCT01703988","author":"Isis Pharmaceuticals","year":"2012"},{"key":"10.1016\/j.addr.2015.03.008_bb0835","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1093\/hmg\/8.1.69","article-title":"Characterization of ATM gene mutations in 66 ataxia telangiectasia families","volume":"8","author":"Sandoval","year":"1999","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2015.03.008_bb0840","doi-asserted-by":"crossref","first-page":"3909","DOI":"10.1073\/pnas.0710865105","article-title":"In vivo correction of a Menkes disease model using antisense oligonucleotides","volume":"105","author":"Madsen","year":"2008","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.addr.2015.03.008_bb0845","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1038\/ng0193-7","article-title":"Isolation of a candidate gene for Menkes disease and evidence that it encodes a copper-transporting ATPase","volume":"3","author":"Vulpe","year":"1993","journal-title":"Nat. Genet."},{"key":"10.1016\/j.addr.2015.03.008_bb0850","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1038\/ejhg.2009.187","article-title":"Menkes disease","volume":"18","author":"Tumer","year":"2010","journal-title":"Eur. J. Hum. Genet."},{"key":"10.1016\/j.addr.2015.03.008_bb0855","doi-asserted-by":"crossref","first-page":"23302","DOI":"10.1074\/jbc.C600143200","article-title":"Stabilization of the tau exon 10 stem loop alters pre-mRNA splicing","volume":"281","author":"Donahue","year":"2006","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.addr.2015.03.008_bb0860","doi-asserted-by":"crossref","first-page":"9836","DOI":"10.1093\/nar\/gks710","article-title":"Targeting a pre-mRNA structure with bipartite antisense molecules modulates tau alternative splicing","volume":"40","author":"Peacey","year":"2012","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0865","doi-asserted-by":"crossref","first-page":"9511","DOI":"10.1021\/bi00198a017","article-title":"Domains of tau protein and interactions with microtubules","volume":"33","author":"Gustke","year":"1994","journal-title":"Biochemistry"},{"key":"10.1016\/j.addr.2015.03.008_bb0870","doi-asserted-by":"crossref","first-page":"702","DOI":"10.1038\/31508","article-title":"Association of missense and 5\u2032-splice-site mutations in tau with the inherited dementia FTDP-17","volume":"393","author":"Hutton","year":"1998","journal-title":"Nature"},{"key":"10.1016\/j.addr.2015.03.008_bb0875","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1089\/nat.2013.0452","article-title":"Preventing formation of toxic N-terminal huntingtin fragments through antisense oligonucleotide-mediated protein modification","volume":"24","author":"Evers","year":"2014","journal-title":"Nucleic Acid Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0880","doi-asserted-by":"crossref","first-page":"783","DOI":"10.1093\/hmg\/7.5.783","article-title":"Truncated N-terminal fragments of huntingtin with expanded glutamine repeats form nuclear and cytoplasmic aggregates in cell culture","volume":"7","author":"Cooper","year":"1998","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2015.03.008_bb0885","doi-asserted-by":"crossref","first-page":"1179","DOI":"10.1016\/j.cell.2006.04.026","article-title":"Cleavage at the caspase-6 site is required for neuronal dysfunction and degeneration due to mutant huntingtin","volume":"125","author":"Graham","year":"2006","journal-title":"Cell"},{"key":"10.1016\/j.addr.2015.03.008_bb0890","doi-asserted-by":"crossref","first-page":"1830","DOI":"10.1523\/JNEUROSCI.21-06-01830.2001","article-title":"Tissue-specific proteolysis of Huntingtin (htt) in human brain: evidence of enhanced levels of N- and C-terminal htt fragments in Huntington's disease striatum","volume":"21","author":"Mende-Mueller","year":"2001","journal-title":"J. Neurosci."},{"key":"10.1016\/j.addr.2015.03.008_bb0895","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1016\/S0169-328X(00)00248-5","article-title":"Antinociceptive effects after intrathecal administration of phosphodiester-, 2\u2032-O-allyl-, and C-5-propyne-modified antisense oligodeoxynucleotides targeting the NMDAR1 subunit in mouse","volume":"86","author":"Rydh-Rinder","year":"2001","journal-title":"Brain Res. Mol. Brain Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0900","doi-asserted-by":"crossref","first-page":"404","DOI":"10.1016\/S0022-3565(25)20148-3","article-title":"Chronic intrathecal infusion of phosphorothioate or phosphodiester antisense oligonucleotides against cytokine responsive gene-2\/IP-10 in experimental allergic encephalomyelitis of lewis rat","volume":"278","author":"Wojcik","year":"1996","journal-title":"J. Pharmacol. Exp. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0905","doi-asserted-by":"crossref","first-page":"193","DOI":"10.1016\/0167-0115(95)00100-P","article-title":"Distribution of phosphodiester and phosphorothioate oligonucleotides in rat brain after intraventricular and intrahippocampal administration determined by in situ hybridization","volume":"59","author":"Yaida","year":"1995","journal-title":"Regul. Pept."},{"key":"10.1016\/j.addr.2015.03.008_bb0910","doi-asserted-by":"crossref","DOI":"10.3171\/foc.1997.3.5.7","article-title":"Distribution and stability of antisense phosphorothioate oligonucleotides in rodent brain following direct intraparenchymal controlled-rate infusion","volume":"3","author":"Broaddus","year":"1997","journal-title":"Neurosurg. Focus."},{"key":"10.1016\/j.addr.2015.03.008_bb0915","doi-asserted-by":"crossref","first-page":"60","DOI":"10.1215\/S1522851705000475","article-title":"Phase 1 trial of a CpG oligodeoxynucleotide for patients with recurrent glioblastoma","volume":"8","author":"Carpentier","year":"2006","journal-title":"Neuro Oncol."},{"key":"10.1016\/j.addr.2015.03.008_bb0920","doi-asserted-by":"crossref","first-page":"94","DOI":"10.1089\/oli.2005.15.94","article-title":"Intracerebral and intrathecal infusion of the TGF-beta 2-specific antisense phosphorothioate oligonucleotide AP 12009 in rabbits and primates: toxicology and safety","volume":"15","author":"Schlingensiepen","year":"2005","journal-title":"Oligonucleotides"},{"key":"10.1016\/j.addr.2015.03.008_bb0925","doi-asserted-by":"crossref","first-page":"906","DOI":"10.1523\/JNEUROSCI.3533-04.2005","article-title":"Inhibition of steroid receptor coactivator-1 blocks estrogen and androgen action on male sex behavior and associated brain plasticity","volume":"25","author":"Charlier","year":"2005","journal-title":"J. Neurosci."},{"key":"10.1016\/j.addr.2015.03.008_bb0930","doi-asserted-by":"crossref","first-page":"119","DOI":"10.1038\/mt.2011.232","article-title":"Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy","volume":"20","author":"Osman","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0935","doi-asserted-by":"crossref","first-page":"854","DOI":"10.1038\/mt.2013.276","article-title":"Dual masking of specific negative splicing regulatory elements resulted in maximal exon 7 inclusion of SMN2 gene","volume":"22","author":"Pao","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.addr.2015.03.008_bb0940","doi-asserted-by":"crossref","first-page":"4127","DOI":"10.1093\/hmg\/ddt261","article-title":"Method for widespread microRNA-155 inhibition prolongs survival in ALS-model mice","volume":"22","author":"Koval","year":"2013","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2015.03.008_bb0945","doi-asserted-by":"crossref","first-page":"4832","DOI":"10.1093\/hmg\/ddu198","article-title":"Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models","volume":"23","author":"Osman","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2015.03.008_bb0950","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1385\/JMN:24:1:123","article-title":"Down-regulation of amyloid precursor protein by peptide nucleic acid in vivo","volume":"24","author":"Boules","year":"2004","journal-title":"J. Mol. Neurosci."},{"key":"10.1016\/j.addr.2015.03.008_bb0955","doi-asserted-by":"crossref","first-page":"752","DOI":"10.1046\/j.1471-4159.2003.02053.x","article-title":"Antisense peptide nucleic acid-mediated knockdown of the p75 neurotrophin receptor delays motor neuron disease in mutant SOD1 transgenic mice","volume":"87","author":"Turner","year":"2003","journal-title":"J. Neurochem."},{"key":"10.1016\/j.addr.2015.03.008_bb0960","series-title":"Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations (SOD-1), ClinicalTrials. gov NCT01041222","author":"Isis Pharmaceuticals","year":"2009"},{"key":"10.1016\/j.addr.2015.03.008_bb0965","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1038\/nature10485","article-title":"Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model","volume":"478","author":"Hua","year":"2011","journal-title":"Nature"},{"key":"10.1016\/j.addr.2015.03.008_bb0970","doi-asserted-by":"crossref","first-page":"845345","DOI":"10.1155\/2013\/845345","article-title":"Presymptomatic treatment with acetylcholinesterase antisense oligonucleotides prolongs survival in ALS (G93A-SOD1) mice","volume":"2013","author":"Marc","year":"2013","journal-title":"Biomed. Res. Int."},{"key":"10.1016\/j.addr.2015.03.008_bb0975","doi-asserted-by":"crossref","first-page":"201","DOI":"10.1001\/jamaneurol.2013.593","article-title":"SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy","volume":"70","author":"Winer","year":"2013","journal-title":"JAMA Neurol."},{"key":"10.1016\/j.addr.2015.03.008_bb0980","doi-asserted-by":"crossref","first-page":"573","DOI":"10.1002\/jnr.20191","article-title":"Antisense peptide nucleic acid targeting GluR3 delays disease onset and progression in the SOD1 G93A mouse model of familial ALS","volume":"77","author":"Rembach","year":"2004","journal-title":"J. Neurosci. Res."},{"key":"10.1016\/j.addr.2015.03.008_bb0985","doi-asserted-by":"crossref","first-page":"217","DOI":"10.3233\/JHD-130057","article-title":"Antisense oligonucleotide-mediated correction of transcriptional dysregulation is correlated with behavioral benefits in the YAC128 mouse model of Huntington's disease","volume":"2","author":"Stanek","year":"2013","journal-title":"J. Huntingtons Dis."},{"key":"10.1016\/j.addr.2015.03.008_bb0990","doi-asserted-by":"crossref","first-page":"387","DOI":"10.1016\/j.freeradbiomed.2013.11.014","article-title":"Antisense oligonucleotide against GSK-3beta in brain of SAMP8 mice improves learning and memory and decreases oxidative stress: involvement of transcription factor Nrf2 and implications for Alzheimer disease","volume":"67","author":"Farr","year":"2014","journal-title":"Free Radic. Biol. Med."}],"container-title":["Advanced Drug Delivery Reviews"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0169409X15000435?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0169409X15000435?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,5,22]],"date-time":"2025-05-22T17:53:18Z","timestamp":1747936398000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0169409X15000435"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2015,6]]},"references-count":198,"alternative-id":["S0169409X15000435"],"URL":"https:\/\/doi.org\/10.1016\/j.addr.2015.03.008","relation":{},"ISSN":["0169-409X"],"issn-type":[{"value":"0169-409X","type":"print"}],"subject":[],"published":{"date-parts":[[2015,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Antisense oligonucleotides in therapy for neurodegenerative disorders","name":"articletitle","label":"Article Title"},{"value":"Advanced Drug Delivery Reviews","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.addr.2015.03.008","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2015 The Authors. Published by Elsevier B.V.","name":"copyright","label":"Copyright"}]}}