{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T19:17:38Z","timestamp":1791919058869,"version":"4.3.4"},"reference-count":175,"publisher":"MDPI AG","issue":"3","license":[{"start":{"date-parts":[[2022,2,2]],"date-time":"2022-02-02T00:00:00Z","timestamp":1643760000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004281","name":"National Science Center","doi-asserted-by":"publisher","award":["2018\/29\/B\/NZ1\/00293"],"award-info":[{"award-number":["2018\/29\/B\/NZ1\/00293"]}],"id":[{"id":"10.13039\/501100004281","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cells"],"abstract":"<jats:p>Polyglutamine (polyQ) diseases, including Huntington\u2019s disease, are a group of late-onset progressive neurological disorders caused by CAG repeat expansions. Although recently, many studies have investigated the pathological features and development of polyQ diseases, many questions remain unanswered. The advancement of new gene-editing technologies, especially the CRISPR-Cas9 technique, has undeniable value for the generation of relevant polyQ models, which substantially support the research process. Here, we review how these tools have been used to correct disease-causing mutations or create isogenic cell lines with different numbers of CAG repeats. We characterize various cellular models such as HEK 293 cells, patient-derived fibroblasts, human embryonic stem cells (hESCs), induced pluripotent stem cells (iPSCs) and animal models generated with the use of genome-editing technology.<\/jats:p>","DOI":"10.3390\/cells11030517","type":"journal-article","created":{"date-parts":[[2022,2,3]],"date-time":"2022-02-03T05:42:33Z","timestamp":1643866953000},"page":"517","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Advances in Modeling Polyglutamine Diseases Using Genome Editing Tools"],"prefix":"10.3390","volume":"11","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1678-6007","authenticated-orcid":false,"given":"Marianna","family":"Karwacka","sequence":"first","affiliation":[{"name":"Department of Genome Engineering, Institute of Bioorganic Chemistry, Polish Academy of Sciences, 61-704 Poznan, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8471-6302","authenticated-orcid":false,"given":"Marta","family":"Olejniczak","sequence":"additional","affiliation":[{"name":"Department of Genome Engineering, Institute of Bioorganic Chemistry, Polish Academy of Sciences, 61-704 Poznan, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2022,2,2]]},"reference":[{"key":"ref_1","first-page":"263","article-title":"Genetics, Mechanisms, and Therapeutic Progress in Polyglutamine Spinocerebellar Ataxias","volume":"16","author":"Buijsen","year":"2019","journal-title":"Neurother. 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