{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T07:48:03Z","timestamp":1790236083250,"version":"4.1.0"},"reference-count":46,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2018,2,26]],"date-time":"2018-02-26T00:00:00Z","timestamp":1519603200000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004281","name":"Narodowe Centrum Nauki","doi-asserted-by":"publisher","award":["2015\/18\/E\/NZ2\/00678"],"award-info":[{"award-number":["2015\/18\/E\/NZ2\/00678"]}],"id":[{"id":"10.13039\/501100004281","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Neurosci."],"DOI":"10.3389\/fnins.2018.00075","type":"journal-article","created":{"date-parts":[[2018,6,19]],"date-time":"2018-06-19T06:00:09Z","timestamp":1529388009000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":103,"title":["Precise Excision of the CAG Tract from the Huntingtin Gene by Cas9 Nickases"],"prefix":"10.3389","volume":"12","author":[{"given":"Magdalena","family":"Dabrowska","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wojciech","family":"Juzwa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wlodzimierz J.","family":"Krzyzosiak","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marta","family":"Olejniczak","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2018,2,26]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","DOI":"10.1371\/currents.hd.0242d2e7ad72225efa72f6964589369a","article-title":"Polyglutamine disease modeling: epitope based screen for homologous recombination using CRISPR\/Cas9 System","volume":"6","author":"An","year":"2014","journal-title":"PLoS Curr"},{"key":"B2","doi-asserted-by":"publisher","first-page":"253","DOI":"10.1016\/j.stem.2012.04.026","article-title":"Genetic correction of huntington's disease phenotypes in induced pluripotent stem cells","volume":"11","author":"An","year":"2012","journal-title":"Cell Stem Cell"},{"key":"B3","doi-asserted-by":"publisher","first-page":"15005","DOI":"10.1038\/nrdp.2015.5","article-title":"Huntington disease","volume":"1","author":"Bates","year":"2015","journal-title":"Nat. Rev. Dis. Prim"},{"key":"B4","doi-asserted-by":"publisher","first-page":"1053","DOI":"10.1038\/mt.2009.17","article-title":"Nonallele-specific silencing of mutant and wild-type Huntingtin demonstrates therapeutic efficacy in Huntington's disease mice","volume":"17","author":"Boudreau","year":"2009","journal-title":"Mol. Ther"},{"key":"B5","doi-asserted-by":"publisher","first-page":"132","DOI":"10.1101\/gr.162339.113","article-title":"Analysis of off-target effects of CRISPR\/Cas-derived RNA-guided endonucleases and nickases","volume":"24","author":"Cho","year":"2014","journal-title":"Genome Res"},{"key":"B6","doi-asserted-by":"publisher","first-page":"853","DOI":"10.1016\/j.molcel.2011.08.017","article-title":"Mechanisms and consequences of alternative polyadenylation","volume":"43","author":"Di Giammartino","year":"2011","journal-title":"Mol. Cell"},{"key":"B7","doi-asserted-by":"publisher","first-page":"387","DOI":"10.1038\/ng0893-387","article-title":"Trinucleotide repeat length instability and age of onset in Huntington's disease","volume":"4","author":"Duyao","year":"1993","journal-title":"Nat. Genet"},{"key":"B8","doi-asserted-by":"publisher","first-page":"860","DOI":"10.1002\/med.21425","article-title":"Discovery of therapeutic approaches for polyglutamine diseases: a summary of recent efforts","volume":"37","author":"Esteves","year":"2017","journal-title":"Med. Res. Rev"},{"key":"B9","doi-asserted-by":"publisher","first-page":"10426","DOI":"10.1093\/nar\/gkt825","article-title":"Self-duplexing CUG repeats selectively inhibit mutant huntingtin expression","volume":"41","author":"Fiszer","year":"2013","journal-title":"Nucleic Acids Res"},{"key":"B10","doi-asserted-by":"publisher","first-page":"6","DOI":"10.1186\/1471-2199-13-6","article-title":"An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases","volume":"13","author":"Fiszer","year":"2012","journal-title":"BMC Mol. Biol"},{"key":"B11","doi-asserted-by":"publisher","first-page":"E3136","DOI":"10.1073\/pnas.1206506109","article-title":"Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6\/2 mice","volume":"109","author":"Garriga-Canut","year":"2012","journal-title":"Proc. Natl. Acad. Sci. U.S.A"},{"key":"B12","doi-asserted-by":"publisher","first-page":"148","DOI":"10.1186\/s13059-016-1012-2","article-title":"Evaluation of off-target and on-target scoring algorithms and integration into the guide RNA selection tool CRISPOR","volume":"17","author":"Haeussler","year":"2016","journal-title":"Genome Biol"},{"key":"B13","doi-asserted-by":"publisher","first-page":"5820","DOI":"10.1073\/pnas.0501507102","article-title":"RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model","volume":"102","author":"Harper","year":"2005","journal-title":"Proc. Natl. Acad. Sci. U.S.A"},{"key":"B14","doi-asserted-by":"publisher","first-page":"28420","DOI":"10.1038\/srep28420","article-title":"Precision modulation of neurodegenerative disease-related gene expression in human iPSC-derived neurons","volume":"6","author":"Heman-Ackah","year":"2016","journal-title":"Sci. Rep"},{"key":"B15","doi-asserted-by":"publisher","first-page":"827","DOI":"10.1038\/nbt.2647","article-title":"DNA targeting specificity of RNA-guided Cas9 nucleases","volume":"31","author":"Hsu","year":"2013","journal-title":"Nat. Biotechnol"},{"key":"B16","doi-asserted-by":"publisher","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":"B17","doi-asserted-by":"publisher","first-page":"32170","DOI":"10.1074\/jbc.M405024200","article-title":"Nonsense mutations in close proximity to the initiation codon fail to trigger full nonsense-mediated mRNA decay","volume":"279","author":"In\u00e1cios","year":"2004","journal-title":"J. Biol. Chem"},{"key":"B18","doi-asserted-by":"publisher","first-page":"816","DOI":"10.1126\/science.1225829","article-title":"A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity","volume":"337","author":"Jinek","year":"2012","journal-title":"Science"},{"key":"B19","doi-asserted-by":"publisher","first-page":"596","DOI":"10.1093\/nar\/24.4.596","article-title":"Transfecting mammalian cells: optimization of critical parameters affecting calcium-phosphate precipitate formation","volume":"24","author":"Jordan","year":"1996","journal-title":"Nucleic Acids Res"},{"key":"B20","doi-asserted-by":"publisher","first-page":"R53","DOI":"10.1093\/hmg\/ddv442","article-title":"Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia","volume":"25","author":"Keiser","year":"2016","journal-title":"Hum. Mol. Genet"},{"key":"B21","doi-asserted-by":"publisher","first-page":"9","DOI":"10.1038\/ng0194-9","article-title":"Unstable expansion of CAG repeat in hereditary dentatorubral-pallidoluysian atrophy (DRPLA)","volume":"6","author":"Koide","year":"1994","journal-title":"Nat. Genet"},{"key":"B22","doi-asserted-by":"publisher","first-page":"754","DOI":"10.3390\/ijms18040754","article-title":"CRISPR-Cas9 mediated gene-silencing of the mutant Huntingtin gene in an in vitro model of Huntington's disease","volume":"18","author":"Kolli","year":"2017","journal-title":"Int. J. Mol. Sci"},{"key":"B23","doi-asserted-by":"publisher","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":"B24","doi-asserted-by":"publisher","first-page":"137","DOI":"10.1016\/j.molcel.2016.02.031","article-title":"Identifying and visualizing functional PAM diversity across CRISPR-cas systems","volume":"62","author":"Leenay","year":"2016","journal-title":"Mol. Cell"},{"key":"B25","doi-asserted-by":"publisher","first-page":"1541","DOI":"10.1093\/hmg\/2.10.1541","article-title":"Differential 3' polyadenylation of the huntington disease gene results in two mRNA species with variable tissue expression","volume":"2","author":"Lin","year":"1993","journal-title":"Hum. Mol. Genet"},{"key":"B26","doi-asserted-by":"publisher","first-page":"833","DOI":"10.1038\/nbt.2675","article-title":"CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering","volume":"31","author":"Mali","year":"2013","journal-title":"Nat. Biotechnol"},{"key":"B27","doi-asserted-by":"publisher","first-page":"2980","DOI":"10.1016\/j.celrep.2017.08.075","article-title":"The self-inactivating KamiCas9 system for the editing of CNS disease genes","volume":"20","author":"Merienne","year":"2017","journal-title":"Cell Rep"},{"key":"B28","doi-asserted-by":"publisher","first-page":"9607","DOI":"10.1073\/pnas.0902420106","article-title":"Zinc-finger directed double-strand breaks within CAG repeat tracts promote repeat instability in human cells","volume":"106","author":"Mittelman","year":"2009","journal-title":"Proc. Natl. Acad. Sci. U.S.A."},{"key":"B29","doi-asserted-by":"publisher","first-page":"12","DOI":"10.1016\/j.ymthe.2016.11.010","article-title":"CRISPR\/Cas9 editing of the mutant huntingtin allele in vitro and in vivo","volume":"25","author":"Monteys","year":"2017","journal-title":"Mol. Ther"},{"key":"B30","doi-asserted-by":"publisher","first-page":"613","DOI":"10.1038\/nrn.2017.92","article-title":"Polyglutamine spinocerebellar ataxias \u2014 from genes to potential treatments","volume":"18","author":"Paulson","year":"2017","journal-title":"Nat. Rev. Neurosci"},{"key":"B31","doi-asserted-by":"publisher","first-page":"905","DOI":"10.3324\/haematol.2010.039206","article-title":"Control of human \u03b2-globin mRNA stability and its impact on beta-thalassemia phenotype","volume":"96","author":"Peixeiro","year":"2011","journal-title":"Haematologica"},{"key":"B32","doi-asserted-by":"publisher","first-page":"1319","DOI":"10.1016\/j.cell.2016.05.053","article-title":"Leveraging rules of nonsense-mediated mRNA decay for genome engineering and personalized medicine","volume":"165","author":"Popp","year":"2016","journal-title":"Cell"},{"key":"B33","doi-asserted-by":"publisher","first-page":"1380","DOI":"10.1016\/j.cell.2013.08.021","article-title":"Double nicking by RNA-guided CRISPR cas9 for enhanced genome editing specificity","volume":"154","author":"Ran","year":"2013","journal-title":"Cell"},{"key":"B34","doi-asserted-by":"publisher","first-page":"e95611","DOI":"10.1371\/journal.pone.0095611","article-title":"Highly specific contractions of a single CAG\/CTG trinucleotide repeat by TALEN in yeast","volume":"9","author":"Richard","year":"2014","journal-title":"PLoS ONE"},{"key":"B35","doi-asserted-by":"publisher","first-page":"3057","DOI":"10.1016\/j.celrep.2017.09.009","article-title":"Alterations in mRNA 3\u2032 UTR isoform abundance accompany gene expression changes in human Huntington's disease brains","volume":"20","author":"Romo","year":"2017","journal-title":"Cell Rep"},{"key":"B36","doi-asserted-by":"publisher","first-page":"910","DOI":"10.1016\/j.neuron.2016.02.003","article-title":"The biology of Huntingtin","volume":"89","author":"Saudou","year":"2016","journal-title":"Neuron"},{"key":"B37","doi-asserted-by":"publisher","first-page":"399","DOI":"10.1038\/nmeth.2857","article-title":"Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects","volume":"11","author":"Shen","year":"2014","journal-title":"Nat. Methods"},{"key":"B38","doi-asserted-by":"publisher","first-page":"4566","DOI":"10.1093\/hmg\/ddw286","article-title":"Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR\/Cas9","volume":"25","author":"Shin","year":"2016","journal-title":"Hum. Mol. Genet."},{"key":"B39","doi-asserted-by":"publisher","first-page":"77","DOI":"10.1038\/352077a0","article-title":"Androgen receptor gene mutations in X-linked spinal and bulbar muscular atrophy","volume":"352","author":"Spada","year":"1991","journal-title":"Nature"},{"key":"B40","doi-asserted-by":"publisher","first-page":"3484","DOI":"10.1093\/emboj\/17.12.3484","article-title":"Binary specification of nonsense codons by splicing and cytoplasmic translation","volume":"17","author":"Thermann","year":"1998","journal-title":"EMBO J"},{"key":"B41","doi-asserted-by":"publisher","first-page":"161","DOI":"10.1016\/B978-0-12-801185-0.00008-8","article-title":"Genome editing using cas9 nickases","volume":"546","author":"Trevino","year":"2014","journal-title":"Methods Enzymol"},{"key":"B42","doi-asserted-by":"publisher","first-page":"24","DOI":"10.1016\/j.ymthe.2016.10.014","article-title":"CRISPR\/Cas9-Induced (CTG,CAG) n repeat instability in the myotonic dystrophy type 1 locus: implications for therapeutic genome editing","volume":"25","author":"Van Agtmaal","year":"2017","journal-title":"Mol. Ther"},{"key":"B43","doi-asserted-by":"publisher","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":"B44","doi-asserted-by":"publisher","first-page":"2719","DOI":"10.1172\/JCI92087","article-title":"CRISPR\/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease","volume":"127","author":"Yang","year":"2017","journal-title":"J. Clin. Invest"},{"key":"B45","doi-asserted-by":"publisher","first-page":"895","DOI":"10.1016\/j.cell.2012.08.002","article-title":"Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression","volume":"150","author":"Yu","year":"2012","journal-title":"Cell"},{"key":"B46","doi-asserted-by":"publisher","first-page":"5405","DOI":"10.1038\/srep05405","article-title":"Comparison of non-canonical PAMs for CRISPR\/Cas9-mediated DNA cleavage in human cells","volume":"4","author":"Zhang","year":"2014","journal-title":"Sci. Rep"}],"container-title":["Frontiers in Neuroscience"],"original-title":[],"link":[{"URL":"http:\/\/journal.frontiersin.org\/article\/10.3389\/fnins.2018.00075\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2018,6,19]],"date-time":"2018-06-19T06:00:15Z","timestamp":1529388015000},"score":1,"resource":{"primary":{"URL":"http:\/\/journal.frontiersin.org\/article\/10.3389\/fnins.2018.00075\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,2,26]]},"references-count":46,"alternative-id":["10.3389\/fnins.2018.00075"],"URL":"https:\/\/doi.org\/10.3389\/fnins.2018.00075","relation":{"has-review":[{"id-type":"doi","id":"10.3410\/f.732845621.793569370","asserted-by":"object"}]},"ISSN":["1662-453X"],"issn-type":[{"value":"1662-453X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018,2,26]]},"article-number":"75"}}