{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,25]],"date-time":"2025-07-25T10:46:00Z","timestamp":1753440360954},"publisher-location":"New York, NY","reference-count":17,"publisher":"Springer New York","isbn-type":[{"type":"print","value":"9781493997831"},{"type":"electronic","value":"9781493997848"}],"license":[{"start":{"date-parts":[[2019,10,5]],"date-time":"2019-10-05T00:00:00Z","timestamp":1570233600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2020]]},"DOI":"10.1007\/978-1-4939-9784-8_17","type":"book-chapter","created":{"date-parts":[[2019,10,4]],"date-time":"2019-10-04T10:03:23Z","timestamp":1570183403000},"page":"269-284","update-policy":"http:\/\/dx.doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":16,"title":["Gene Therapy for Huntington\u2019s Disease Using Targeted Endonucleases"],"prefix":"10.1007","author":[{"given":"Magdalena","family":"Dabrowska","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marta","family":"Olejniczak","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2019,10,5]]},"reference":[{"key":"17_CR1","doi-asserted-by":"publisher","DOI":"10.1038\/nrdp.2015.5","volume":"1","author":"GP Bates","year":"2015","unstructured":"Bates GP, Dorsey R, Gusella JF et al (2015) Huntington disease. Nat Rev Dis Primers 1:15005. \n                  https:\/\/doi.org\/10.1038\/nrdp.2015.5","journal-title":"Nat Rev Dis Primers"},{"key":"17_CR2","doi-asserted-by":"publisher","first-page":"837","DOI":"10.1016\/S1474-4422(17)30280-6","volume":"16","author":"EJ Wild","year":"2017","unstructured":"Wild EJ, Tabrizi SJ (2017) Therapies targeting DNA and RNA in Huntington\u2019s disease. Lancet Neurol 16:837\u2013847. \n                  https:\/\/doi.org\/10.1016\/S1474-4422(17)30280-6","journal-title":"Lancet Neurol"},{"key":"17_CR3","doi-asserted-by":"publisher","first-page":"671","DOI":"10.1016\/j.molcel.2016.12.016","volume":"65","author":"A Shibata","year":"2017","unstructured":"Shibata A, Steinlage M, Barton O et al (2017) DNA double-strand break resection occurs during non-homologous end joining in G1 but is distinct from resection during homologous recombination. Mol Cell 65:671\u2013684.e5. \n                  https:\/\/doi.org\/10.1016\/j.molcel.2016.12.016","journal-title":"Mol Cell"},{"key":"17_CR4","doi-asserted-by":"publisher","first-page":"132","DOI":"10.1101\/gr.162339.113","volume":"24","author":"SW Cho","year":"2014","unstructured":"Cho SW, Kim S, Kim Y et al (2014) Analysis of off-target effects of CRISPR\/Cas-derived RNA-guided endonucleases and nickases. Genome Res 24:132\u2013141. \n                  https:\/\/doi.org\/10.1101\/gr.162339.113","journal-title":"Genome Res"},{"key":"17_CR5","doi-asserted-by":"publisher","first-page":"1380","DOI":"10.1016\/j.cell.2013.08.021","volume":"154","author":"FA Ran","year":"2013","unstructured":"Ran FA, Hsu PD, Lin CY et al (2013) Double nicking by RNA-guided CRISPR cas9 for enhanced genome editing specificity. Cell 154:1380\u20131389. \n                  https:\/\/doi.org\/10.1016\/j.cell.2013.08.021","journal-title":"Cell"},{"key":"17_CR6","doi-asserted-by":"publisher","first-page":"2585","DOI":"10.1016\/j.ymthe.2017.08.015","volume":"25","author":"T Kocher","year":"2017","unstructured":"Kocher T, Peking P, Klausegger A et al (2017) Cut and paste: efficient homology-directed repair of a dominant negative KRT14 Mutation via CRISPR\/Cas9 nickases. Mol Ther 25:2585\u20132598. \n                  https:\/\/doi.org\/10.1016\/j.ymthe.2017.08.015","journal-title":"Mol Ther"},{"key":"17_CR7","doi-asserted-by":"publisher","unstructured":"Eggenschwiler R, Moslem M, Fr\u00e1guas MS et al (2016) Improved bi-allelic modification of a transcriptionally silent locus in patient-derived iPSC by Cas9 nickase. Nat Publ Gr:1\u201314. \n                  https:\/\/doi.org\/10.1038\/srep38198","DOI":"10.1038\/srep38198"},{"key":"17_CR8","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1007\/s00294-018-0865-1","volume":"65","author":"V Mosbach","year":"2019","unstructured":"Mosbach V, Poggi L, Richard G-F (2019) Trinucleotide repeat instability during double-strand break repair: from mechanisms to gene therapy. Curr Genet 65:17\u201328. \n                  https:\/\/doi.org\/10.1007\/s00294-018-0865-1","journal-title":"Curr Genet"},{"issue":"20","key":"17_CR9","doi-asserted-by":"publisher","first-page":"4566","DOI":"10.1093\/hmg\/ddw286","volume":"25","author":"JW Shin","year":"2016","unstructured":"Shin JW, Kim K-H, Chao MJ et al (2016) Permanent inactivation of Huntington\u2019s disease mutation by personalized allele-specific CRISPR\/Cas9. Hum Mol Genet 25(20):4566\u20134576. \n                  https:\/\/doi.org\/10.1093\/hmg\/ddw286","journal-title":"Hum Mol Genet"},{"key":"17_CR10","doi-asserted-by":"publisher","first-page":"12","DOI":"10.1016\/j.ymthe.2016.11.010","volume":"25","author":"AM Monteys","year":"2017","unstructured":"Monteys AM, Ebanks SA, Keiser MS, Davidson BL (2017) CRISPR\/Cas9 editing of the mutant Huntingtin Allele in vitro and in vivo. Mol Ther 25:12\u201323. \n                  https:\/\/doi.org\/10.1016\/j.ymthe.2016.11.010","journal-title":"Mol Ther"},{"key":"17_CR11","doi-asserted-by":"publisher","first-page":"2719","DOI":"10.1172\/JCI92087","volume":"127","author":"S Yang","year":"2017","unstructured":"Yang S, Chang R, Yang H et al (2017) CRISPR\/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington\u2019s disease. J Clin Invest 127:2719\u20132724. \n                  https:\/\/doi.org\/10.1172\/JCI92087","journal-title":"J Clin Invest"},{"key":"17_CR12","doi-asserted-by":"publisher","first-page":"75","DOI":"10.3389\/FNINS.2018.00075","volume":"12","author":"M Dabrowska","year":"2018","unstructured":"Dabrowska M, Juzwa W, Krzyzosiak WJ, Olejniczak M (2018) Precise excision of the CAG tract from the Huntingtin gene by Cas9 nickases. Front Neurosci 12:75. \n                  https:\/\/doi.org\/10.3389\/FNINS.2018.00075","journal-title":"Front Neurosci"},{"key":"17_CR13","doi-asserted-by":"publisher","first-page":"15587","DOI":"10.1038\/srep15587","volume":"5","author":"MK Ramlee","year":"2015","unstructured":"Ramlee MK, Yan T, Cheung AMS et al (2015) High-throughput genotyping of CRISPR\/Cas9-mediated mutants using fluorescent PCR-capillary gel electrophoresis. Sci Rep 5:15587. \n                  https:\/\/doi.org\/10.1038\/srep15587","journal-title":"Sci Rep"},{"key":"17_CR14","doi-asserted-by":"publisher","first-page":"148","DOI":"10.1186\/s13059-016-1012-2","volume":"17","author":"M Haeussler","year":"2016","unstructured":"Haeussler M, Sch\u00f6nig K, Eckert H et al (2016) Evaluation of off-target and on-target scoring algorithms and integration into the guide RNA selection tool CRISPOR. Genome Biol 17:148. \n                  https:\/\/doi.org\/10.1186\/s13059-016-1012-2","journal-title":"Genome Biol"},{"key":"17_CR15","doi-asserted-by":"publisher","first-page":"455","DOI":"10.1007\/s12539-018-0298-z","volume":"10","author":"Y Cui","year":"2018","unstructured":"Cui Y, Xu J, Cheng M et al (2018) Review of CRISPR\/Cas9 sgRNA design tools. Interdiscip Sci Comput Life Sci 10:455\u2013465. \n                  https:\/\/doi.org\/10.1007\/s12539-018-0298-z","journal-title":"Interdiscip Sci Comput Life Sci"},{"key":"17_CR16","doi-asserted-by":"publisher","first-page":"29","DOI":"10.1016\/j.ymeth.2017.05.009","volume":"121\u2013122","author":"S Yumlu","year":"2017","unstructured":"Yumlu S, Stumm J, Bashir S et al (2017) Gene editing and clonal isolation of human induced pluripotent stem cells using CRISPR\/Cas9. Methods 121\u2013122:29\u201344. \n                  https:\/\/doi.org\/10.1016\/j.ymeth.2017.05.009","journal-title":"Methods"},{"key":"17_CR17","doi-asserted-by":"publisher","first-page":"e101","DOI":"10.1093\/nar\/gky505","volume":"46","author":"M Dabrowska","year":"2018","unstructured":"Dabrowska M, Czubak K, Juzwa W et al (2018) qEva-CRISPR: a method for quantitative evaluation of CRISPR\/Cas-mediated genome editing in target and off-target sites. Nucleic Acids Res 46:e101. \n                  https:\/\/doi.org\/10.1093\/nar\/gky505","journal-title":"Nucleic Acids Res"}],"container-title":["Methods in Molecular Biology","Trinucleotide Repeats"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/978-1-4939-9784-8_17","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,10,4]],"date-time":"2019-10-04T10:07:35Z","timestamp":1570183655000},"score":1,"resource":{"primary":{"URL":"http:\/\/link.springer.com\/10.1007\/978-1-4939-9784-8_17"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,10,5]]},"ISBN":["9781493997831","9781493997848"],"references-count":17,"URL":"https:\/\/doi.org\/10.1007\/978-1-4939-9784-8_17","relation":{},"ISSN":["1064-3745","1940-6029"],"issn-type":[{"type":"print","value":"1064-3745"},{"type":"electronic","value":"1940-6029"}],"subject":[],"published":{"date-parts":[[2019,10,5]]},"assertion":[{"value":"5 October 2019","order":1,"name":"first_online","label":"First Online","group":{"name":"ChapterHistory","label":"Chapter History"}}]}}