{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T10:33:07Z","timestamp":1793183587092,"version":"4.5.0"},"reference-count":39,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"funder":[{"DOI":"10.13039\/501100001691","name":"Japan Society for the Promotion of Science","doi-asserted-by":"publisher","award":["19H00924"],"award-info":[{"award-number":["19H00924"]}],"id":[{"id":"10.13039\/501100001691","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001691","name":"Japan Society for the Promotion of Science","doi-asserted-by":"publisher","award":["22H00351"],"award-info":[{"award-number":["22H00351"]}],"id":[{"id":"10.13039\/501100001691","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001691","name":"Japan Society for the Promotion of Science","doi-asserted-by":"publisher","award":["20H02880"],"award-info":[{"award-number":["20H02880"]}],"id":[{"id":"10.13039\/501100001691","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Bioorganic & Medicinal Chemistry Letters"],"published-print":{"date-parts":[[2023,1]]},"DOI":"10.1016\/j.bmcl.2022.129082","type":"journal-article","created":{"date-parts":[[2022,11,19]],"date-time":"2022-11-19T02:16:22Z","timestamp":1668824182000},"page":"129082","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":3,"special_numbering":"C","title":["A small molecule binding to TGGAA pentanucleotide repeats that cause spinocerebellar ataxia type 31"],"prefix":"10.1016","volume":"79","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3653-0383","authenticated-orcid":false,"given":"Tomonori","family":"Shibata","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1705-5265","authenticated-orcid":false,"given":"Kazuhiko","family":"Nakatani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.bmcl.2022.129082_b0005","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1038\/nrg1689","article-title":"Repeat instability: mechanisms of dynamic mutations","volume":"6","author":"Pearson","year":"2005","journal-title":"Nat Rev Genet"},{"key":"10.1016\/j.bmcl.2022.129082_b0010","doi-asserted-by":"crossref","first-page":"932","DOI":"10.1038\/nature05977","article-title":"Expandable DNA repeats and human disease","volume":"447","author":"Mirkin","year":"2007","journal-title":"Nature"},{"key":"10.1016\/j.bmcl.2022.129082_b0015","doi-asserted-by":"crossref","first-page":"e101112","DOI":"10.15252\/embj.2018101112","article-title":"RNA toxicity in non-coding repeat expansion disorders","volume":"39","author":"Swinnen","year":"2020","journal-title":"EMBO J"},{"key":"10.1016\/j.bmcl.2022.129082_b0020","doi-asserted-by":"crossref","first-page":"652","DOI":"10.1038\/nchembio.416","article-title":"Replication-dependent instability at (CTG)\u00b7(CAG) repeat hairpins in human cells","volume":"6","author":"Liu","year":"2010","journal-title":"Nat Chem Biol"},{"key":"10.1016\/j.bmcl.2022.129082_b0025","doi-asserted-by":"crossref","first-page":"1618","DOI":"10.1128\/MCB.06727-11","article-title":"Altered replication in human cells promotes DMPK (CTG)n\u00b7(CAG)n repeat instability","volume":"32","author":"Liu","year":"2012","journal-title":"Mol Cell Biol"},{"key":"10.1016\/j.bmcl.2022.129082_b0030","doi-asserted-by":"crossref","first-page":"669","DOI":"10.1038\/s41594-018-0094-9","article-title":"Mechanisms of genetic instability caused by (CGG)n repeats in an experimental mammalian system","volume":"25","author":"Kononenko","year":"2018","journal-title":"Nat Struct Mol Biol"},{"key":"10.1016\/j.bmcl.2022.129082_b0035","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1038\/nsmb1042","article-title":"Transcription promotes contraction of CAG repeat tracts in human cells","volume":"13","author":"Lin","year":"2006","journal-title":"Nat Struct Mol Biol"},{"key":"10.1016\/j.bmcl.2022.129082_b0040","doi-asserted-by":"crossref","first-page":"692","DOI":"10.1073\/pnas.0909740107","article-title":"R loops stimulate genetic instability of CTG\u00b7CAG repeats","volume":"107","author":"Lin","year":"2010","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1016\/j.bmcl.2022.129082_b0045","doi-asserted-by":"crossref","first-page":"580","DOI":"10.1093\/hmg\/ddq501","article-title":"Bidirectional transcription stimulates expansion and contraction of expanded (CTG)\u2022(CAG) repeats","volume":"20","author":"Nakamori","year":"2011","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.bmcl.2022.129082_b0050","doi-asserted-by":"crossref","first-page":"6209","DOI":"10.1128\/MCB.00739-07","article-title":"Transcription-induced CAG repeat contraction in human cells is mediated in part by transcription-coupled nucleotide excision repair","volume":"27","author":"Lin","year":"2007","journal-title":"Mol Cell Biol"},{"key":"10.1016\/j.bmcl.2022.129082_b0055","doi-asserted-by":"crossref","first-page":"471","DOI":"10.1038\/70598","article-title":"Msh2 deficiency prevents in vivo somatic instability of the CAG repeat in Huntington disease transgenic mice","volume":"23","author":"Manley","year":"1999","journal-title":"Nat Genet"},{"key":"10.1016\/j.bmcl.2022.129082_b0060","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1038\/86906","article-title":"Trinucleotide expansion in haploid germ cells by gap repair","volume":"27","author":"Kovtun","year":"2001","journal-title":"Nat Genet"},{"key":"10.1016\/j.bmcl.2022.129082_b0065","doi-asserted-by":"crossref","first-page":"2264","DOI":"10.1093\/emboj\/cdg202","article-title":"CTG repeat instability and size variation timing in DNA repair-deficient mice","volume":"22","author":"Savouret","year":"2003","journal-title":"EMBO J"},{"key":"10.1016\/j.bmcl.2022.129082_b0070","doi-asserted-by":"crossref","first-page":"29958","DOI":"10.1074\/jbc.M112.356758","article-title":"DNA mismatch repair complex MutS\u03b2 promotes GAA\u00b7TTC repeat expansion in human cells","volume":"287","author":"Halabi","year":"2012","journal-title":"J Biol Chem"},{"key":"10.1016\/j.bmcl.2022.129082_b0075","first-page":"7087","article-title":"Muts\u03b2 generates both expansions and contractions in a mouse model of the Fragile X-associated disorders","volume":"24","author":"Zhao","year":"2015","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.bmcl.2022.129082_b0080","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1016\/j.sbi.2006.05.004","article-title":"DNA structures, repeat expansions and human hereditary disorders","volume":"16","author":"Mirkin","year":"2006","journal-title":"Curr Opin Struct Biol"},{"key":"10.1016\/j.bmcl.2022.129082_b0085","doi-asserted-by":"crossref","first-page":"16673","DOI":"10.1038\/srep16673","article-title":"Topology of a G-quadruplex DNA formed by C9orf72 hexanucleotide repeats associated with ALS and FTD","volume":"5","author":"Zhou","year":"2015","journal-title":"Sci Rep"},{"key":"10.1016\/j.bmcl.2022.129082_b0090","doi-asserted-by":"crossref","first-page":"17944","DOI":"10.1038\/srep17944","article-title":"Anti-sense DNA d(GGCCCC)n expansions in C9ORF72 form i-motifs and protonated hairpins","volume":"5","author":"Kovanda","year":"2015","journal-title":"Sci Rep"},{"key":"10.1016\/j.bmcl.2022.129082_b0095","doi-asserted-by":"crossref","first-page":"4534","DOI":"10.1093\/nar\/gkf572","article-title":"Slipped-strand DNAs formed by long (CAG)\u00b7(CTG) repeats: slipped-out repeats and slip-out junctions","volume":"30","author":"Pearson","year":"2002","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.bmcl.2022.129082_b0100","doi-asserted-by":"crossref","first-page":"e1003866","DOI":"10.1371\/journal.pgen.1003866","article-title":"Detection of slipped-DNAs at the trinucleotide repeats of the myotonic dystrophy type I disease locus in patient tissues","volume":"9","author":"Axford","year":"2013","journal-title":"PLoS Genet"},{"key":"10.1016\/j.bmcl.2022.129082_b0105","doi-asserted-by":"crossref","first-page":"544","DOI":"10.1016\/j.ajhg.2009.09.019","article-title":"Spinocerebellar Ataxia Type 31 Is associated with \u201cinserted\u201d penta-nucleotide repeats containing (TGGAA)n","volume":"85","author":"Sato","year":"2009","journal-title":"Am J Hum Genet"},{"key":"10.1016\/j.bmcl.2022.129082_b0110","doi-asserted-by":"crossref","first-page":"600","DOI":"10.1111\/neup.12032","article-title":"Abnormal RNA structures (RNA foci) containing a penta-nucleotide repeat (UGGAA)n in the Purkinje cell nucleus is associated with spinocerebellar ataxia type 31 pathogenesis","volume":"33","author":"Niimi","year":"2013","journal-title":"Neuropathology"},{"key":"10.1016\/j.bmcl.2022.129082_b0115","doi-asserted-by":"crossref","first-page":"108","DOI":"10.1016\/j.neuron.2017.02.046","article-title":"Regulatory role of RNA chaperone TDP-43 for RNA misfolding and repeat-associated translation in SCA31","volume":"94","author":"Ishiguro","year":"2017","journal-title":"Neuron"},{"key":"10.1016\/j.bmcl.2022.129082_b0120","doi-asserted-by":"crossref","first-page":"9535","DOI":"10.1073\/pnas.1708691114","article-title":"Parity-dependent hairpin configurations of repetitive DNA sequence promote slippage associated with DNA expansion","volume":"114","author":"Huang","year":"2017","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1016\/j.bmcl.2022.129082_b0125","doi-asserted-by":"crossref","first-page":"1055","DOI":"10.1246\/bcsj.82.1055","article-title":"Recognition of Mismatched base pairs in DNA","volume":"82","author":"Nakatani","year":"2009","journal-title":"Bull Chem Soc Jpn"},{"key":"10.1016\/j.bmcl.2022.129082_b0130","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1038\/nchembio708","article-title":"Small-molecule ligand induces nucleotide flipping in (CAG)n trinucleotide repeats","volume":"1","author":"Nakatani","year":"2005","journal-title":"Nat Chem Biol"},{"key":"10.1016\/j.bmcl.2022.129082_b0135","doi-asserted-by":"crossref","first-page":"7280","DOI":"10.1002\/anie.200502282","article-title":"Binding of naphthyridine carbamate dimer to the (CGG)n repeat results in the disruption of the G-C base pairing","volume":"44","author":"Peng","year":"2005","journal-title":"Angew Chemie - Int Ed"},{"key":"10.1016\/j.bmcl.2022.129082_b0140","doi-asserted-by":"crossref","first-page":"4646","DOI":"10.1016\/j.bmc.2014.07.019","article-title":"Sequence-specific DNA alkylation and transcriptional inhibition by long-chain hairpin pyrrole-imidazole polyamide-chlorambucil conjugates targeting CAG\/CTG trinucleotide repeats","volume":"22","author":"Asamitsu","year":"2014","journal-title":"Bioorganic Med Chem"},{"key":"10.1016\/j.bmcl.2022.129082_b0145","doi-asserted-by":"crossref","first-page":"14180","DOI":"10.1021\/jacs.5b09266","article-title":"Rationally designed small molecules that target both the DNA and RNA causing myotonic dystrophy type 1","volume":"137","author":"Nguyen","year":"2015","journal-title":"J Am Chem Soc"},{"key":"10.1016\/j.bmcl.2022.129082_b0150","doi-asserted-by":"crossref","first-page":"8761","DOI":"10.1002\/anie.201703989","article-title":"Induced-fit recognition of CCG Trinucleotide repeats by a nickel-chromomycin complex resulting in large-scale DNA deformation","volume":"56","author":"Tseng","year":"2017","journal-title":"Angew Chemie - Int Ed"},{"key":"10.1016\/j.bmcl.2022.129082_b0155","doi-asserted-by":"crossref","first-page":"2978","DOI":"10.1016\/j.bmc.2019.05.022","article-title":"Development of novel macrocyclic small molecules that target CTG trinucleotide repeats","volume":"27","author":"Serrano","year":"2019","journal-title":"Bioorganic Med Chem"},{"key":"10.1016\/j.bmcl.2022.129082_b0160","doi-asserted-by":"crossref","first-page":"11165","DOI":"10.1021\/jacs.0c03591","article-title":"Structural basis for targeting T: T mismatch with triaminotriazine-acridine conjugate induces a U-shaped head-to-head four-way junction in CTG repeat DNA","volume":"142","author":"Chien","year":"2020","journal-title":"J Am Chem Soc"},{"key":"10.1016\/j.bmcl.2022.129082_b0165","first-page":"gkab227","article-title":"Targeting the ALS\/FTD-associated A-DNA kink with anthracene-based metal complex causes DNA backbone straightening and groove contraction","author":"Jhan","year":"2021","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.bmcl.2022.129082_b0170","doi-asserted-by":"crossref","first-page":"10906","DOI":"10.1093\/nar\/gkz832","article-title":"Structural insights into synthetic ligands targeting A-A pairs in disease-related CAG RNA repeats","volume":"47","author":"Mukherjee","year":"2019","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.bmcl.2022.129082_b0175","doi-asserted-by":"crossref","first-page":"14305","DOI":"10.1002\/chem.202001572","article-title":"The dimeric form of 1,3-diaminoisoquinoline derivative rescued the Mis-splicing of Atp2a1 and Clcn1 genes in myotonic dystrophy type 1 mouse model","volume":"26","author":"Matsumoto","year":"2020","journal-title":"Chem. - A Eur. J."},{"key":"10.1016\/j.bmcl.2022.129082_b0180","doi-asserted-by":"crossref","first-page":"236","DOI":"10.1038\/s41467-020-20487-4","article-title":"Small molecule targeting r(UGGAA)n disrupts RNA foci and alleviates disease phenotype in Drosophila model","volume":"12","author":"Shibata","year":"2021","journal-title":"Nat Commun"},{"key":"10.1016\/j.bmcl.2022.129082_b0185","doi-asserted-by":"crossref","first-page":"146","DOI":"10.1038\/s41588-019-0575-8","article-title":"A slipped-CAG DNA-binding small molecule induces trinucleotide-repeat contractions in vivo","volume":"52","author":"Nakamori","year":"2020","journal-title":"Nat Genet"},{"key":"10.1016\/j.bmcl.2022.129082_b0190","doi-asserted-by":"crossref","first-page":"9621","DOI":"10.1093\/nar\/gkac740","article-title":"NMR determination of the 2:1 binding complex of naphthyridine carbamate dimer (NCD) and CGG\/CGG triad in double-stranded DNA","volume":"50","author":"Yamada","year":"2022","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.bmcl.2022.129082_b0195","doi-asserted-by":"crossref","first-page":"12650","DOI":"10.1021\/ja0109186","article-title":"Recognition of guanine-guanine mismatches by the dimeric form of 2-amino-1,8-naphthyridine","volume":"123","author":"Nakatani","year":"2001","journal-title":"J Am Chem Soc"}],"container-title":["Bioorganic & Medicinal Chemistry Letters"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0960894X22005583?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0960894X22005583?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,18]],"date-time":"2025-10-18T07:46:45Z","timestamp":1760773605000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0960894X22005583"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,1]]},"references-count":39,"alternative-id":["S0960894X22005583"],"URL":"https:\/\/doi.org\/10.1016\/j.bmcl.2022.129082","relation":{},"ISSN":["0960-894X"],"issn-type":[{"value":"0960-894X","type":"print"}],"subject":[],"published":{"date-parts":[[2023,1]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"A small molecule binding to TGGAA pentanucleotide repeats that cause spinocerebellar ataxia type 31","name":"articletitle","label":"Article Title"},{"value":"Bioorganic & Medicinal Chemistry Letters","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.bmcl.2022.129082","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 Elsevier Ltd. All rights reserved.","name":"copyright","label":"Copyright"}],"article-number":"129082"}}