{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T18:30:04Z","timestamp":1792175404051,"version":"4.4.0"},"reference-count":63,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2023,6,1]],"date-time":"2023-06-01T00:00:00Z","timestamp":1685577600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,6,1]],"date-time":"2023-06-01T00:00:00Z","timestamp":1685577600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,5,11]],"date-time":"2023-05-11T00:00:00Z","timestamp":1683763200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100005725","name":"CHDI Foundation","doi-asserted-by":"publisher","award":["CHDI-6367"],"award-info":[{"award-number":["CHDI-6367"]}],"id":[{"id":"10.13039\/100005725","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005725","name":"CHDI Foundation","doi-asserted-by":"publisher","award":["R35 GM131839"],"award-info":[{"award-number":["R35 GM131839"]}],"id":[{"id":"10.13039\/100005725","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005725","name":"CHDI Foundation","doi-asserted-by":"publisher","award":["S10 OD020012"],"award-info":[{"award-number":["S10 OD020012"]}],"id":[{"id":"10.13039\/100005725","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005614","name":"Biogen","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005614","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100016012","name":"Huntington's Disease Society of America","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100016012","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["CHDI-6367"],"award-info":[{"award-number":["CHDI-6367"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["U01 NS114098"],"award-info":[{"award-number":["U01 NS114098"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["R35 GM131839"],"award-info":[{"award-number":["R35 GM131839"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["S10 OD020012"],"award-info":[{"award-number":["S10 OD020012"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["R01 NS106245"],"award-info":[{"award-number":["R01 NS106245"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2023,6]]},"DOI":"10.1016\/j.ymthe.2023.05.006","type":"journal-article","created":{"date-parts":[[2023,5,11]],"date-time":"2023-05-11T21:48:45Z","timestamp":1683841725000},"page":"1661-1674","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":65,"title":["Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington\u2019s disease"],"prefix":"10.1016","volume":"31","author":[{"given":"Daniel","family":"O'Reilly","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jillian","family":"Belgrad","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chantal","family":"Ferguson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashley","family":"Summers","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ellen","family":"Sapp","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cassandra","family":"McHugh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ella","family":"Mathews","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adel","family":"Boudi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julianna","family":"Buchwald","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Socheata","family":"Ly","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dimas","family":"Moreno","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raymond","family":"Furgal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric","family":"Luu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zachary","family":"Kennedy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vignesh","family":"Hariharan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn","family":"Monopoli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"X. William","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffery","family":"Carroll","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marian","family":"DiFiglia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neil","family":"Aronin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6928-8071","authenticated-orcid":false,"given":"Anastasia","family":"Khvorova","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2023.05.006_bib1","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1038\/s41582-020-0389-4","article-title":"Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities","volume":"16","author":"Tabrizi","year":"2020","journal-title":"Nat. Rev. Neurol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib2","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1016\/S1474-4422(17)30280-6","article-title":"Therapies targeting DNA and RNA in Huntington's disease","volume":"16","author":"Wild","year":"2017","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib3","doi-asserted-by":"crossref","first-page":"645","DOI":"10.1016\/S1474-4422(22)00121-1","article-title":"Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities","volume":"21","author":"Tabrizi","year":"2022","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib4","series-title":"Polyglutamine Disorders","first-page":"1","article-title":"Clinical features of huntington\u2019s disease","author":"Ghosh","year":"2018"},{"key":"10.1016\/j.ymthe.2023.05.006_bib5","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1111\/ene.13413","article-title":"Huntington's disease: a clinical review","volume":"25","author":"McColgan","year":"2018","journal-title":"Eur. J. Neurol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib6","doi-asserted-by":"crossref","first-page":"632","DOI":"10.1016\/S1474-4422(22)00120-X","article-title":"A biological classification of Huntington's disease: the Integrated Staging System","volume":"21","author":"Tabrizi","year":"2022","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib7","doi-asserted-by":"crossref","first-page":"7","DOI":"10.3233\/JHD-200429","article-title":"The contribution of somatic expansion of the CAG repeat to symptomatic development in huntington\u2019s disease: a historical perspective","volume":"10","author":"Monckton","year":"2021","journal-title":"J.\u00a0Huntingtons Dis."},{"key":"10.1016\/j.ymthe.2023.05.006_bib8","doi-asserted-by":"crossref","first-page":"3039","DOI":"10.1093\/hmg\/ddp242","article-title":"Somatic expansion of the Huntington's disease CAG repeat in the brain is associated with an earlier age of disease onset","volume":"18","author":"Swami","year":"2009","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2023.05.006_bib9","doi-asserted-by":"crossref","first-page":"1562","DOI":"10.1128\/MCB.20.5.1562-1570.2000","article-title":"Terminally differentiated human neurons repair transcribed genes but display attenuated global DNA repair and modulation of repair gene expression","volume":"20","author":"Nouspikel","year":"2000","journal-title":"Mol. Cell. Biol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib10","doi-asserted-by":"crossref","first-page":"1193","DOI":"10.1016\/0896-6273(95)90106-X","article-title":"CAG expansion affects the expression of mutant Huntingtin in the Huntington's disease brain","volume":"15","author":"Aronin","year":"1995","journal-title":"Neuron"},{"key":"10.1016\/j.ymthe.2023.05.006_bib11","doi-asserted-by":"crossref","first-page":"204","DOI":"10.1038\/s41467-020-20426-3","article-title":"Conformational and migrational dynamics of slipped-strand DNA three-way junctions containing trinucleotide repeats","volume":"12","author":"Hu","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2023.05.006_bib12","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1016\/S0014-5793(98)00022-2","article-title":"Slipped loop structure of DNA: a specific nucleotide sequence forms only one unique conformer","volume":"422","author":"Khomyakova","year":"1998","journal-title":"FEBS Lett."},{"key":"10.1016\/j.ymthe.2023.05.006_bib13","doi-asserted-by":"crossref","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.ymthe.2023.05.006_bib14","doi-asserted-by":"crossref","first-page":"887","DOI":"10.1016\/j.cell.2019.06.036","article-title":"CAG repeat not polyglutamine length determines timing of huntington\u2019s disease onset","volume":"178","author":"Lee","year":"2019","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2023.05.006_bib15","doi-asserted-by":"crossref","first-page":"1876","DOI":"10.1093\/brain\/awz115","article-title":"MSH3 modifies somatic instability and disease severity in Huntington\u2019s and myotonic dystrophy type 1","volume":"142","author":"Flower","year":"2019","journal-title":"Brain."},{"key":"10.1016\/j.ymthe.2023.05.006_bib16","doi-asserted-by":"crossref","first-page":"701","DOI":"10.1016\/S1474-4422(17)30161-8","article-title":"Identification of genetic variants associated with Huntington's disease progression: a genome-wide association study","volume":"16","author":"Moss","year":"2017","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib17","doi-asserted-by":"crossref","first-page":"60","DOI":"10.1016\/j.dnarep.2019.03.009","article-title":"The mismatch repair-dependent DNA damage response: mechanisms and implications","volume":"78","author":"Gupta","year":"2019","journal-title":"DNA Repair"},{"key":"10.1016\/j.ymthe.2023.05.006_bib18","doi-asserted-by":"crossref","first-page":"122","DOI":"10.3389\/fmolb.2020.00122","article-title":"Mismatch repair pathway, genome stability and cancer","volume":"7","author":"Pe\u0107ina-\u0160laus","year":"2020","journal-title":"Front. Mol. Biosci."},{"key":"10.1016\/j.ymthe.2023.05.006_bib19","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1016\/j.ajhg.2016.06.015","article-title":"Exome sequencing identifies biallelic MSH3 germline Mutations as a recessive subtype of colorectal adenomatous polyposis","volume":"99","author":"Adam","year":"2016","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.ymthe.2023.05.006_bib20","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1080\/10409238.2021.1881433","article-title":"Coordinated roles of SLX4 and MutS\u03b2 in DNA repair and the maintenance of genome stability","volume":"56","author":"Young","year":"2021","journal-title":"Crit. Rev. Biochem. Mol. Biol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib21","doi-asserted-by":"crossref","first-page":"458","DOI":"10.1186\/s12864-021-07793-x","article-title":"Online database for brain cancer-implicated genes: exploring the subtype-specific mechanisms of brain cancer","volume":"22","author":"Zhao","year":"2021","journal-title":"BMC Genomics"},{"key":"10.1016\/j.ymthe.2023.05.006_bib22","first-page":"A10","article-title":"C01 the effect of mismatch repair proteins in a huntington\u2019s disease cellular model","volume":"92","author":"Stone","year":"2021","journal-title":"J.\u00a0Neurol. Neurosurg. Psychiatry"},{"key":"10.1016\/j.ymthe.2023.05.006_bib23","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1016\/j.nbd.2008.09.014","article-title":"Intergenerational and striatal CAG repeat instability in Huntington's disease knock-in mice involve different DNA repair genes","volume":"33","author":"Dragileva","year":"2009","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.ymthe.2023.05.006_bib24","doi-asserted-by":"crossref","DOI":"10.1016\/j.bcp.2021.114432","article-title":"The growth of siRNA-based therapeutics: updated clinical studies","volume":"189","author":"Zhang","year":"2021","journal-title":"Biochem. Pharmacol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib25","doi-asserted-by":"crossref","first-page":"101","DOI":"10.1038\/s41392-020-0207-x","article-title":"Therapeutic siRNA: state of the art","volume":"5","author":"Hu","year":"2020","journal-title":"Signal Transduct. Target. Ther."},{"key":"10.1016\/j.ymthe.2023.05.006_bib26","doi-asserted-by":"crossref","first-page":"2185","DOI":"10.1093\/nar\/gky037","article-title":"Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in\u00a0vivo","volume":"46","author":"Hassler","year":"2018","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2023.05.006_bib27","doi-asserted-by":"crossref","first-page":"5126","DOI":"10.1039\/D0CS01430C","article-title":"Recent progress in non-native nucleic acid modifications","volume":"50","author":"McKenzie","year":"2021","journal-title":"Chem. Soc. Rev."},{"key":"10.1016\/j.ymthe.2023.05.006_bib28","doi-asserted-by":"crossref","first-page":"937","DOI":"10.1016\/j.chembiol.2012.07.011","article-title":"Designing chemically modified oligonucleotides for targeted gene silencing","volume":"19","author":"Deleavey","year":"2012","journal-title":"Chem. Biol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib29","doi-asserted-by":"crossref","first-page":"238","DOI":"10.1038\/nbt.3765","article-title":"The chemical evolution of oligonucleotide therapies of clinical utility","volume":"35","author":"Khvorova","year":"2017","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib30","doi-asserted-by":"crossref","first-page":"421","DOI":"10.1038\/s41573-019-0017-4","article-title":"The current state and future directions of RNAi-based therapeutics","volume":"18","author":"Setten","year":"2019","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.ymthe.2023.05.006_bib31","doi-asserted-by":"crossref","first-page":"991","DOI":"10.1016\/j.omtn.2020.07.029","article-title":"Single-stranded phosphorothioated regions enhance cellular uptake of cholesterol-conjugated siRNA but not silencing efficacy","volume":"21","author":"Ly","year":"2020","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.ymthe.2023.05.006_bib32","doi-asserted-by":"crossref","first-page":"884","DOI":"10.1038\/s41587-019-0205-0","article-title":"A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system","volume":"37","author":"Alterman","year":"2019","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib33","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.152203","article-title":"Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep","volume":"6","author":"Ferguson","year":"2021","journal-title":"JCI Insight"},{"key":"10.1016\/j.ymthe.2023.05.006_bib34","doi-asserted-by":"crossref","first-page":"10905","DOI":"10.1093\/nar\/gky745","article-title":"Functional features defining the efficacy of cholesterol-conjugated, self-deliverable, chemically modified siRNAs","volume":"46","author":"Shmushkovich","year":"2018","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2023.05.006_bib35","doi-asserted-by":"crossref","first-page":"7581","DOI":"10.1093\/nar\/gkx507","article-title":"5\u0384-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo","volume":"45","author":"Haraszti","year":"2017","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2023.05.006_bib36","doi-asserted-by":"crossref","first-page":"2709","DOI":"10.1016\/j.ymthe.2022.04.019","article-title":"RNAi-based modulation of IFN-\u03b3 signaling in skin","volume":"30","author":"Tang","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2023.05.006_bib37","doi-asserted-by":"crossref","first-page":"384","DOI":"10.1016\/j.omtn.2020.06.006","article-title":"Cell type impacts accessibility of mRNA to silencing by RNA interference","volume":"21","author":"Ferguson","year":"2020","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.ymthe.2023.05.006_bib38","doi-asserted-by":"crossref","first-page":"3907","DOI":"10.1093\/nar\/gkab152","article-title":"Somatic CAG expansion in Huntington's disease is dependent on the MLH3 endonuclease domain, which can be excluded via splice redirection","volume":"49","author":"Roy","year":"2021","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2023.05.006_bib39","doi-asserted-by":"crossref","first-page":"2799","DOI":"10.1093\/hmg\/9.19.2799","article-title":"Dominant phenotypes produced by the HD mutation in STHdhQ111 striatal cells","volume":"9","author":"Trettel","year":"2000","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2023.05.006_bib40","article-title":"Quantification of age-dependent somatic CAG repeat instability in Hdh CAG knock-in mice reveals different expansion dynamics in striatum and liver","volume":"6","author":"Lee","year":"2011","journal-title":"PLoS One"},{"key":"10.1016\/j.ymthe.2023.05.006_bib41","doi-asserted-by":"crossref","first-page":"115","DOI":"10.1093\/hmg\/8.1.115","article-title":"Length-dependent gametic CAG repeat instability in the huntington's disease knock-in mouse","volume":"8","author":"Wheeler","year":"1999","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2023.05.006_bib42","doi-asserted-by":"crossref","first-page":"734","DOI":"10.1021\/acs.jmedchem.7b01147","article-title":"Facile synthesis, geometry, and 2\u2032-substituent-dependent in\u00a0vivo activity of 5\u2032-(E)- and 5\u2032-(Z)-Vinylphosphonate-Modified siRNA conjugates","volume":"61","author":"Parmar","year":"2018","journal-title":"J.\u00a0Med. Chem."},{"key":"10.1016\/j.ymthe.2023.05.006_bib43","doi-asserted-by":"crossref","first-page":"e266","DOI":"10.1038\/mtna.2015.38","article-title":"Hydrophobically modified siRNAs silence huntingtin mRNA in primary neurons and mouse brain","volume":"4","author":"Alterman","year":"2015","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.ymthe.2023.05.006_bib44","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1186\/1752-0509-4-29","article-title":"A novel approach to investigate tissue-specific trinucleotide repeat instability","volume":"4","author":"Lee","year":"2010","journal-title":"BMC Syst. Biol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib45","doi-asserted-by":"crossref","first-page":"1173","DOI":"10.1016\/j.neuron.2022.01.006","article-title":"Uninterrupted CAG repeat drives striatum-selective transcriptionopathy and nuclear pathogenesis in human Huntingtin BAC mice","volume":"110","author":"Gu","year":"2022","journal-title":"Neuron"},{"key":"10.1016\/j.ymthe.2023.05.006_bib46","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1200\/PO.17.00073","article-title":"Landscape of microsatellite instability across 39 cancer types","volume":"2017","author":"Bonneville","year":"2017","journal-title":"JCO Precis. Oncol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib47","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1002\/mc.20146","article-title":"Use of mononucleotide repeat markers for detection of microsatellite instability in mouse tumors","volume":"44","author":"Bacher","year":"2005","journal-title":"Mol. Carcinog."},{"key":"10.1016\/j.ymthe.2023.05.006_bib48","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1038\/d41586-021-01177-7","article-title":"Failure of genetic therapies for Huntington\u2019s devastates community","volume":"593","author":"Kwon","year":"2021","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2023.05.006_bib49","doi-asserted-by":"crossref","first-page":"801","DOI":"10.1016\/j.neuron.2019.01.039","article-title":"Huntingtin lowering strategies for disease modification in huntington's disease","volume":"101","author":"Tabrizi","year":"2019","journal-title":"Neuron"},{"key":"10.1016\/j.ymthe.2023.05.006_bib50","doi-asserted-by":"crossref","DOI":"10.1016\/j.celrep.2021.109649","article-title":"FAN1 controls mismatch repair complex assembly via MLH1 retention to stabilize CAG repeat expansion in Huntington's disease","volume":"36","author":"Goold","year":"2021","journal-title":"Cell Rep."},{"key":"10.1016\/j.ymthe.2023.05.006_bib51","article-title":"Silencing of ApoE with divalent siRNAs drives activation of immune clearance pathways and improves amyloid pathology in mouse models of Alzheimer\u2019s Disease","author":"Ferguson","year":"2022","journal-title":"bioRxiv"},{"key":"10.1016\/j.ymthe.2023.05.006_bib52","article-title":"Huntingtin lowering reduces somatic instability at CAG-expanded loci","author":"Coffey","year":"2020","journal-title":"bioRxiv"},{"key":"10.1016\/j.ymthe.2023.05.006_bib53","doi-asserted-by":"crossref","first-page":"1032","DOI":"10.1016\/j.molcel.2019.12.029","article-title":"Directed RNase H cleavage of nascent transcripts causes transcription termination","volume":"77","author":"Lai","year":"2020","journal-title":"Mol. Cell"},{"key":"10.1016\/j.ymthe.2023.05.006_bib54","doi-asserted-by":"crossref","first-page":"e00029-20","DOI":"10.1128\/MCB.00029-20","article-title":"The human DNA mismatch repair protein MSH3 contains nuclear localization and export signals that enable nuclear-cytosolic shuttling in response to inflammation","volume":"40","author":"Tseng-Rogenski","year":"2020","journal-title":"Mol. Cell. Biol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib55","doi-asserted-by":"crossref","first-page":"5107","DOI":"10.1158\/1078-0432.CCR-10-0821","article-title":"Therapeutic targeting of the DNA mismatch repair pathway","volume":"16","author":"Martin","year":"2010","journal-title":"Clin. Cancer Res."},{"key":"10.1016\/j.ymthe.2023.05.006_bib56","doi-asserted-by":"crossref","first-page":"228","DOI":"10.1007\/s100380070031","article-title":"Association between single nucleotide polymorphisms in the hMSH3 gene and sporadic colon cancer with microsatellite instability","volume":"45","author":"Orimo","year":"2000","journal-title":"J.\u00a0Hum. Genet."},{"key":"10.1016\/j.ymthe.2023.05.006_bib57","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pgen.1003930","article-title":"Mismatch repair genes Mlh1 and Mlh3 modify CAG instability in huntington's disease mice: genome-wide and candidate approaches","volume":"9","author":"Pinto","year":"2013","journal-title":"Plos Genet."},{"key":"10.1016\/j.ymthe.2023.05.006_bib58","doi-asserted-by":"crossref","first-page":"3859","DOI":"10.1093\/hmg\/ddx286","article-title":"A modifier of Huntington's disease onset at the MLH1 locus","volume":"26","author":"Lee","year":"2017","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2023.05.006_bib59","doi-asserted-by":"crossref","first-page":"128","DOI":"10.1089\/nat.2018.0725","article-title":"Improving siRNA delivery in\u00a0vivo through lipid conjugation","volume":"28","author":"Osborn","year":"2018","journal-title":"Nucleic Acid Ther."},{"key":"10.1016\/j.ymthe.2023.05.006_bib60","doi-asserted-by":"crossref","first-page":"1500","DOI":"10.1038\/s41587-022-01334-x","article-title":"Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates","volume":"40","author":"Brown","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2023.05.006_bib61","doi-asserted-by":"crossref","first-page":"887","DOI":"10.1016\/j.cell.2019.06.036","article-title":"CAG repeat not polyglutamine length determines timing of huntington\u2019s disease onset","volume":"178","year":"2019","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2023.05.006_bib62","doi-asserted-by":"crossref","first-page":"1075","DOI":"10.1016\/0896-6273(95)90346-1","article-title":"Huntingtin is a cytoplasmic protein associated with vesicles in human and rat brain neurons","volume":"14","author":"DiFiglia","year":"1995","journal-title":"Neuron"},{"key":"10.1016\/j.ymthe.2023.05.006_bib63","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2020.104950","article-title":"Protein changes in synaptosomes of Huntington's disease knock-in mice are dependent on age and brain region","volume":"141","author":"Sapp","year":"2020","journal-title":"Neurobiol. Dis."}],"updated-by":[{"DOI":"10.1016\/j.ymthe.2023.09.016","type":"erratum","label":"Erratum","source":"publisher","updated":{"date-parts":[[2023,11,1]],"date-time":"2023-11-01T00:00:00Z","timestamp":1698796800000}}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001623002629?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001623002629?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,5,12]],"date-time":"2026-05-12T20:06:17Z","timestamp":1778616377000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001623002629"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,6]]},"references-count":63,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2023,6]]}},"alternative-id":["S1525001623002629"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2023.05.006","relation":{"has-preprint":[{"id-type":"doi","id":"10.1101\/2022.09.06.506795","asserted-by":"object"}]},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2023,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington\u2019s disease","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2023.05.006","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 The Author(s).","name":"copyright","label":"Copyright"}]}}