{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T17:43:59Z","timestamp":1793036639881,"version":"4.5.0"},"reference-count":57,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2025,6,1]],"date-time":"2025-06-01T00:00:00Z","timestamp":1748736000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,6,1]],"date-time":"2025-06-01T00:00:00Z","timestamp":1748736000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,4,15]],"date-time":"2025-04-15T00:00:00Z","timestamp":1744675200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004348","name":"Region Stockholm","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004348","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001858","name":"Sweden's Innovation Agency","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100001858","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100018713","name":"Center for Innovative Medicine","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100018713","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003792","name":"Brain Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003792","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010665","name":"EU Framework Programme for Research and Innovation Marie Sk\u0142odowska-Curie Actions","doi-asserted-by":"publisher","award":["956070"],"award-info":[{"award-number":["956070"]}],"id":[{"id":"10.13039\/100010665","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100009708","name":"Novo Nordisk Foundation","doi-asserted-by":"publisher","award":["NNF21OC0072778"],"award-info":[{"award-number":["NNF21OC0072778"]}],"id":[{"id":"10.13039\/501100009708","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000090","name":"US Office of Congressionally Directed Medical Research Programs","doi-asserted-by":"publisher","award":["HT9425-23-1-0337"],"award-info":[{"award-number":["HT9425-23-1-0337"]}],"id":[{"id":"10.13039\/100000090","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004359","name":"Swedish Research Council","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004359","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100002108","name":"Friedreich's Ataxia Research Alliance","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100002108","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010661","name":"Horizon 2020 Framework Programme","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100010661","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100011747","name":"University of Copenhagen Novo Nordisk Foundation Center for Basic Metabolic Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100011747","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Nucleic Acids"],"published-print":{"date-parts":[[2025,6]]},"DOI":"10.1016\/j.omtn.2025.102541","type":"journal-article","created":{"date-parts":[[2025,4,17]],"date-time":"2025-04-17T13:09:17Z","timestamp":1744895357000},"page":"102541","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":6,"title":["Anti-gene oligonucleotides targeting Friedreich\u2019s ataxia expanded GAA\u22c5TTC repeats increase Frataxin expression"],"prefix":"10.1016","volume":"36","author":[{"given":"Negin","family":"Mozafari","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Salom\u00e9","family":"Milagres","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tea","family":"Umek","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina S.J.","family":"Rocha","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia M.","family":"Vargiu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fiona","family":"Freyberger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Osama","family":"Saher","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marek","family":"Napierala","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jill S.","family":"Napierala","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pontus","family":"Blomberg","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Per T.","family":"J\u00f8rgensen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tanel","family":"Punga","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C. I. Edvard","family":"Smith","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesper","family":"Wengel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8327-846X","authenticated-orcid":false,"given":"Rula","family":"Zain","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtn.2025.102541_bib1","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.jns.2011.01.010","article-title":"Friedreich's ataxia: pathology, pathogenesis, and molecular genetics","volume":"303","author":"Koeppen","year":"2011","journal-title":"J. Neurol. Sci."},{"key":"10.1016\/j.omtn.2025.102541_bib2","doi-asserted-by":"crossref","DOI":"10.3390\/ijms22041815","article-title":"Future Prospects of Gene Therapy for Friedreich's Ataxia","volume":"22","author":"Ocana-Santero","year":"2021","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.omtn.2025.102541_bib3","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1007\/s00415-009-1002-3","article-title":"Friedreich ataxia: the clinical picture","volume":"256","author":"Pandolfo","year":"2009","journal-title":"J. Neurol."},{"key":"10.1016\/j.omtn.2025.102541_bib4","doi-asserted-by":"crossref","first-page":"86","DOI":"10.1007\/s004399900201","article-title":"Genotype and phenotype analysis of Friedreich's ataxia compound heterozygous patients","volume":"106","author":"De Castro","year":"2000","journal-title":"Hum. Genet."},{"key":"10.1016\/j.omtn.2025.102541_bib5","doi-asserted-by":"crossref","first-page":"1423","DOI":"10.1126\/science.271.5254.1423","article-title":"Friedreich's ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansion","volume":"271","author":"Campuzano","year":"1996","journal-title":"Science (New York, NY)"},{"key":"10.1016\/j.omtn.2025.102541_bib6","doi-asserted-by":"crossref","first-page":"200","DOI":"10.1002\/1531-8249(199902)45:2<200::AID-ANA10>3.0.CO;2-U","article-title":"Friedreich's ataxia: point mutations and clinical presentation of compound heterozygotes","volume":"45","author":"Coss\u00e9e","year":"1999","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omtn.2025.102541_bib7","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2019.104606","article-title":"Friedreich ataxia- pathogenesis and implications for therapies","volume":"132","author":"Delatycki","year":"2019","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtn.2025.102541_bib8","doi-asserted-by":"crossref","first-page":"1771","DOI":"10.1093\/hmg\/6.11.1771","article-title":"Frataxin is reduced in Friedreich ataxia patients and is associated with mitochondrial membranes","volume":"6","author":"Campuzano","year":"1997","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2025.102541_bib9","doi-asserted-by":"crossref","first-page":"9276","DOI":"10.1074\/jbc.RA119.007716","article-title":"Mechanism of frataxin \"bypass\" in human iron-sulfur cluster biosynthesis with implications for Friedreich's ataxia","volume":"294","author":"Das","year":"2019","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.omtn.2025.102541_bib10","doi-asserted-by":"crossref","first-page":"36773","DOI":"10.1074\/jbc.M113.525857","article-title":"Frataxin directly stimulates mitochondrial cysteine desulfurase by exposing substrate-binding sites, and a mutant Fe-S cluster scaffold protein with frataxin-bypassing ability acts similarly","volume":"288","author":"Pandey","year":"2013","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.omtn.2025.102541_bib11","doi-asserted-by":"crossref","first-page":"129","DOI":"10.1016\/j.pjnns.2018.02.003","article-title":"Progress in the treatment of Friedreich ataxia","volume":"52","author":"Tai","year":"2018","journal-title":"Neurol. Neurochir. Pol."},{"key":"10.1016\/j.omtn.2025.102541_bib12","doi-asserted-by":"crossref","first-page":"684","DOI":"10.1089\/hum.2013.180","article-title":"Cell and gene therapy for Friedreich ataxia: progress to date","volume":"25","author":"Evans-Galea","year":"2014","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtn.2025.102541_bib13","doi-asserted-by":"crossref","first-page":"14588","DOI":"10.1074\/jbc.273.23.14588","article-title":"Inhibitory effects of expanded GAA.TTC triplet repeats from intron I of the Friedreich ataxia gene on transcription and replication in vivo","volume":"273","author":"Ohshima","year":"1998","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.omtn.2025.102541_bib14","doi-asserted-by":"crossref","first-page":"11497","DOI":"10.1073\/pnas.0604939103","article-title":"DNA sequence-specific polyamides alleviate transcription inhibition associated with long GAA.TTC repeats in Friedreich's ataxia","volume":"103","author":"Burnett","year":"2006","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2025.102541_bib15","first-page":"1251","article-title":"Atypical Friedreich ataxia caused by compound heterozygosity for a novel missense mutation and the GAA triplet-repeat expansion","volume":"60","author":"Bidichandani","year":"1997","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.omtn.2025.102541_bib16","doi-asserted-by":"crossref","first-page":"396","DOI":"10.1002\/(SICI)1096-8628(19981012)79:5<396::AID-AJMG13>3.0.CO;2-M","article-title":"Identification of a missense mutation in a Friedreich's ataxia patient: implications for diagnosis and carrier studies","volume":"79","author":"Bartolo","year":"1998","journal-title":"Am. J. Med. Genet."},{"key":"10.1016\/j.omtn.2025.102541_bib17","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1002\/ana.21052","article-title":"Progressive GAA expansions in dorsal root ganglia of Friedreich's ataxia patients","volume":"61","author":"De Biase","year":"2007","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omtn.2025.102541_bib18","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0189990","article-title":"Somatic instability of the expanded GAA repeats in Friedreich's ataxia","volume":"12","author":"Long","year":"2017","journal-title":"PLoS One"},{"key":"10.1016\/j.omtn.2025.102541_bib19","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.ygeno.2007.04.001","article-title":"Somatic instability of the expanded GAA triplet-repeat sequence in Friedreich ataxia progresses throughout life","volume":"90","author":"De Biase","year":"2007","journal-title":"Genomics"},{"key":"10.1016\/j.omtn.2025.102541_bib20","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1002\/emmm.201000064","article-title":"Long intronic GAA repeats causing Friedreich ataxia impede transcription elongation","volume":"2","author":"Punga","year":"2010","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtn.2025.102541_bib21","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1080\/21541264.2015.1026538","article-title":"Friedreich's ataxia\u2013a case of aberrant transcription termination?","volume":"6","author":"Butler","year":"2015","journal-title":"Transcription"},{"key":"10.1016\/j.omtn.2025.102541_bib22","doi-asserted-by":"crossref","first-page":"735","DOI":"10.1093\/hmg\/ddm346","article-title":"The Friedreich ataxia GAA repeat expansion mutation induces comparable epigenetic changes in human and transgenic mouse brain and heart tissues","volume":"17","author":"Al-Mahdawi","year":"2008","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2025.102541_bib23","doi-asserted-by":"crossref","first-page":"4209","DOI":"10.1074\/jbc.M110.194035","article-title":"Repeat expansion affects both transcription initiation and elongation in friedreich ataxia cells","volume":"286","author":"Kumari","year":"2011","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.omtn.2025.102541_bib24","first-page":"6932","article-title":"Expanded GAA repeats impede transcription elongation through the FXN gene and induce transcriptional silencing that is restricted to the FXN locus","volume":"24","author":"Li","year":"2015","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2025.102541_bib25","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1146\/annurev.bb.23.060194.002545","article-title":"H-DNA and related structures","volume":"23","author":"Mirkin","year":"1994","journal-title":"Annu. Rev. Biophys. Biomol. Struct."},{"key":"10.1016\/j.omtn.2025.102541_bib26","doi-asserted-by":"crossref","first-page":"2087","DOI":"10.1002\/j.1460-2075.1989.tb03617.x","article-title":"Structural polymorphism of homopurine--homopyrimidine sequences: the secondary DNA structure adopted by a d(GA.CT)22 sequence in the presence of zinc ions","volume":"8","author":"Bernu\u00e9s","year":"1989","journal-title":"EMBO J."},{"key":"10.1016\/j.omtn.2025.102541_bib27","doi-asserted-by":"crossref","first-page":"4067","DOI":"10.1093\/nar\/18.14.4067","article-title":"DNA-sequence and metal-ion specificity of the formation of \u2217H-DNA","volume":"18","author":"Bernu\u00e9s","year":"1990","journal-title":"Nucleic. Acids. Res."},{"key":"10.1016\/j.omtn.2025.102541_bib28","doi-asserted-by":"crossref","first-page":"3538","DOI":"10.1016\/j.molcel.2022.08.007","article-title":"S1-END-seq reveals DNA secondary structures in human cells","volume":"82","author":"Matos-Rodrigues","year":"2022","journal-title":"Mol. Cell"},{"key":"10.1016\/j.omtn.2025.102541_bib29","doi-asserted-by":"crossref","first-page":"1645","DOI":"10.2147\/JMDH.S292945","article-title":"Friedreich Ataxia: Multidisciplinary Clinical Care","volume":"14","author":"Lynch","year":"2021","journal-title":"J. Multidiscip. Healthc."},{"key":"10.1016\/j.omtn.2025.102541_bib30","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1080\/13543784.2023.2173063","article-title":"Omaveloxolone: an activator of Nrf2 for the treatment of Friedreich ataxia","volume":"32","author":"Profeta","year":"2023","journal-title":"Expet Opin. Invest. Drugs"},{"key":"10.1016\/j.omtn.2025.102541_bib31","doi-asserted-by":"crossref","first-page":"551","DOI":"10.1038\/nchembio815","article-title":"Histone deacetylase inhibitors reverse gene silencing in Friedreich's ataxia","volume":"2","author":"Herman","year":"2006","journal-title":"Nat. Chem. Biol."},{"key":"10.1016\/j.omtn.2025.102541_bib32","doi-asserted-by":"crossref","first-page":"489","DOI":"10.1002\/ana.24260","article-title":"Epigenetic therapy for Friedreich ataxia","volume":"76","author":"Soragni","year":"2014","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omtn.2025.102541_bib33","doi-asserted-by":"crossref","first-page":"504","DOI":"10.1016\/S0140-6736(14)60382-2","article-title":"Epigenetic and neurological effects and safety of high-dose nicotinamide in patients with Friedreich's ataxia: an exploratory, open-label, dose-escalation study","volume":"384","author":"Libri","year":"2014","journal-title":"Lancet (London, England)"},{"key":"10.1016\/j.omtn.2025.102541_bib34","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1038\/gt.2016.89","article-title":"Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich ataxia","volume":"24","author":"Ouellet","year":"2017","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtn.2025.102541_bib35","doi-asserted-by":"crossref","first-page":"11560","DOI":"10.1093\/nar\/gkab954","article-title":"Targeting 3' and 5' untranslated regions with antisense oligonucleotides to stabilize frataxin mRNA and increase protein expression","volume":"49","author":"Li","year":"2021","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2025.102541_bib36","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1089\/nat.2017.0703","article-title":"Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat","volume":"28","author":"Li","year":"2018","journal-title":"Nucleic Acid Therapeut."},{"key":"10.1016\/j.omtn.2025.102541_bib37","article-title":"Activating frataxin expression by repeat-targeted nucleic acids","volume":"7","author":"Li","year":"2016","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtn.2025.102541_bib38","doi-asserted-by":"crossref","first-page":"364","DOI":"10.1080\/15476286.2022.2043650","article-title":"Difficulties translating antisense-mediated activation of Frataxin expression from cell culture to mice","volume":"19","author":"Kilikevicius","year":"2022","journal-title":"RNA Biol."},{"key":"10.1016\/j.omtn.2025.102541_bib39","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0165788","article-title":"Disruption of Higher Order DNA Structures in Friedreich's Ataxia (GAA)n Repeats by PNA or LNA Targeting","volume":"11","author":"Bergquist","year":"2016","journal-title":"PLoS One"},{"key":"10.1016\/j.omtn.2025.102541_bib40","doi-asserted-by":"crossref","first-page":"2629","DOI":"10.1002\/cbic.200900263","article-title":"Structure-specific recognition of Friedreich's ataxia (GAA)n repeats by benzoquinoquinoxaline derivatives","volume":"10","author":"Bergquist","year":"2009","journal-title":"Chembiochem"},{"key":"10.1016\/j.omtn.2025.102541_bib41","doi-asserted-by":"crossref","first-page":"8532","DOI":"10.1093\/nar\/gkad441","article-title":"Large-scale expansions of Friedreich's ataxia GAATTC repeats in an experimental human system: role of DNA replication and prevention by LNA-DNA oligonucleotides and PNA oligomers","volume":"51","author":"Rastokina","year":"2023","journal-title":"Nucleic acids research"},{"key":"10.1016\/j.omtn.2025.102541_bib42","doi-asserted-by":"crossref","first-page":"5974","DOI":"10.1021\/ja012288d","article-title":"Locked nucleic acid (LNA) recognition of RNA: NMR solution structures of LNA:RNA hybrids","volume":"124","author":"Petersen","year":"2002","journal-title":"J. Am. Chem. Soc."},{"key":"10.1016\/j.omtn.2025.102541_bib43","doi-asserted-by":"crossref","first-page":"3257","DOI":"10.1093\/nar\/gkt007","article-title":"Development of bis-locked nucleic acid (bisLNA) oligonucleotides for efficient invasion of supercoiled duplex DNA","volume":"41","author":"Moreno","year":"2013","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2025.102541_bib44","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1089\/nat.2022.0046","article-title":"Chemical Modifications and Design Influence the Potency of Huntingtin Anti-Gene Oligonucleotides","volume":"33","author":"Saher","year":"2023","journal-title":"Nucleic Acid Therapeut."},{"key":"10.1016\/j.omtn.2025.102541_bib45","doi-asserted-by":"crossref","DOI":"10.1093\/nar\/gkv626","article-title":"Ca2+ enrichment in culture medium potentiates effect of oligonucleotides","volume":"43","author":"Hori","year":"2015","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2025.102541_bib46","doi-asserted-by":"crossref","first-page":"1169","DOI":"10.1056\/NEJM199610173351601","article-title":"Clinical and genetic abnormalities in patients with Friedreich's ataxia","volume":"335","author":"D\u00fcrr","year":"1996","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102541_bib47","doi-asserted-by":"crossref","first-page":"475","DOI":"10.1038\/333475a0","article-title":"Chemical probing of homopurine-homopyrimidine mirror repeats in supercoiled DNA","volume":"333","author":"Voloshin","year":"1988","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2025.102541_bib48","doi-asserted-by":"crossref","first-page":"2815","DOI":"10.1093\/nar\/28.14.2815","article-title":"The GAA\u2217TTC triplet repeat expanded in Friedreich's ataxia impedes transcription elongation by T7 RNA polymerase in a length and supercoil dependent manner","volume":"28","author":"Grabczyk","year":"2000","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2025.102541_bib49","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1016\/B978-0-12-407676-1.00002-0","article-title":"Biological activity and biotechnological aspects of locked nucleic acids","volume":"82","author":"Lundin","year":"2013","journal-title":"Adv. Genet."},{"key":"10.1016\/j.omtn.2025.102541_bib50","doi-asserted-by":"crossref","DOI":"10.3389\/fneur.2021.736253","article-title":"Friedreich's Ataxia Frequency in a Large Cohort of Genetically Undetermined Ataxia Patients","volume":"12","author":"Brown","year":"2021","journal-title":"Front. Neurol."},{"key":"10.1016\/j.omtn.2025.102541_bib51","doi-asserted-by":"crossref","first-page":"367","DOI":"10.1016\/j.omtm.2022.02.002","article-title":"In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency","volume":"24","author":"Huichalaf","year":"2022","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtn.2025.102541_bib52","doi-asserted-by":"crossref","first-page":"749","DOI":"10.1016\/j.chembiol.2003.09.014","article-title":"Recognition of chromosomal DNA by PNAs","volume":"11","author":"Kaihatsu","year":"2004","journal-title":"Chem. Biol."},{"key":"10.1016\/j.omtn.2025.102541_bib53","doi-asserted-by":"crossref","first-page":"11103","DOI":"10.1093\/nar\/gkz963","article-title":"New insights into protein-DNA binding specificity from hydrogen bond based comparative study","volume":"47","author":"Lin","year":"2019","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2025.102541_bib54","doi-asserted-by":"crossref","first-page":"5153","DOI":"10.1093\/nar\/gkx111","article-title":"CTG repeat-targeting oligonucleotides for down-regulating Huntingtin expression","volume":"45","author":"Zaghloul","year":"2017","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2025.102541_bib55","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1089\/nat.2021.0021","article-title":"Oligonucleotides Targeting DNA Repeats Downregulate Huntingtin Gene Expression in Huntington's Patient-Derived Neural Model System","volume":"31","author":"Umek","year":"2021","journal-title":"Nucleic Acid Therapeut."},{"key":"10.1016\/j.omtn.2025.102541_bib56","doi-asserted-by":"crossref","first-page":"248","DOI":"10.1007\/s13311-019-00712-9","article-title":"Targeted Oligonucleotides for Treating Neurodegenerative Tandem Repeat Diseases","volume":"16","author":"Zain","year":"2019","journal-title":"Neurotherapeutics"},{"key":"10.1016\/j.omtn.2025.102541_bib57","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/j.bbadis.2007.11.006","article-title":"The effects of frataxin silencing in HeLa cells are rescued by the expression of human mitochondrial ferritin","volume":"1782","author":"Zanella","year":"2008","journal-title":"Biochim. Biophys. Acta."}],"container-title":["Molecular Therapy Nucleic Acids"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253125000952?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253125000952?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,17]],"date-time":"2025-11-17T04:46:30Z","timestamp":1763354790000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2162253125000952"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,6]]},"references-count":57,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2025,6]]}},"alternative-id":["S2162253125000952"],"URL":"https:\/\/doi.org\/10.1016\/j.omtn.2025.102541","relation":{"has-preprint":[{"id-type":"doi","id":"10.1101\/2024.01.25.577034","asserted-by":"object"}]},"ISSN":["2162-2531"],"issn-type":[{"value":"2162-2531","type":"print"}],"subject":[],"published":{"date-parts":[[2025,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Anti-gene oligonucleotides targeting Friedreich\u2019s ataxia expanded GAA\u22c5TTC repeats increase Frataxin expression","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Nucleic Acids","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtn.2025.102541","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Authors. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"102541"}}