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Here, we present a precise and programmable method called prime editor\u2013mediated correction of nucleotide repeat expansion (PE-CORE) for correcting pathogenic nucleotide repeat expansion. PE-CORE leverages a prime editor and paired pegRNAs to achieve targeted correction of repeat sequences. We demonstrate the effectiveness of PE-CORE in HEK293T cells and patient-derived induced pluripotent stem cells (iPSCs). Specifically, we focus on spinal and bulbar muscular atrophy and spinocerebellar ataxia type, two diseases associated with nucleotide repeat expansion. Our results demonstrate the successful correction of pathogenic expansions in iPSCs and subsequent differentiation into motor neurons. Specifically, we detect distinct downshifts in the size of both the mRNA and protein, confirming the functional correction of the iPSC-derived motor neurons. These findings highlight PE-CORE as a precision tool for addressing the intricate challenges of nucleotide repeat expansion disorders, paving the way for targeted therapies and potential clinical applications.<\/jats:p>","DOI":"10.1093\/nar\/gkae310","type":"journal-article","created":{"date-parts":[[2024,4,25]],"date-time":"2024-04-25T11:55:25Z","timestamp":1714046125000},"page":"5792-5803","source":"Crossref","is-referenced-by-count":11,"title":["Precise editing of pathogenic nucleotide repeat expansions in iPSCs using paired prime editor"],"prefix":"10.1093","volume":"52","author":[{"given":"Hye-Yeon","family":"Hwang","sequence":"first","affiliation":[{"name":"Department of Precision Medicine, Sungkyunkwan University School of Medicine , Suwon\u00a0 16419 , Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dongmin","family":"Gim","sequence":"additional","affiliation":[{"name":"School of Pharmacy, Sungkyunkwan University , Suwon\u00a0 16419 , Republic of Korea"},{"name":"Department of Biopharmaceutical Convergence, Sungkyunkwan University , Suwon\u00a0 16419 , Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hwalin","family":"Yi","sequence":"additional","affiliation":[{"name":"Department of Precision Medicine, Sungkyunkwan University School of Medicine , Suwon\u00a0 16419 , Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hyewon","family":"Jung","sequence":"additional","affiliation":[{"name":"School of Pharmacy, Sungkyunkwan University , Suwon\u00a0 16419 , Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jaecheol","family":"Lee","sequence":"additional","affiliation":[{"name":"School of Pharmacy, Sungkyunkwan University , Suwon\u00a0 16419 , Republic of Korea"},{"name":"Department of Biopharmaceutical Convergence, Sungkyunkwan University , Suwon\u00a0 16419 , Republic of Korea"},{"name":"Biomedical Institute for Convergence at SKKU (BICS), Sungkyunkwan University , Suwon\u00a0 16419 , Republic of Korea"},{"name":"Department of Biohealth Regulatory Science, Sungkyunkwan University , Suwon\u00a0 16419 , Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0806-5979","authenticated-orcid":false,"given":"Daesik","family":"Kim","sequence":"additional","affiliation":[{"name":"Department of Precision Medicine, Sungkyunkwan University School of Medicine , Suwon\u00a0 16419 , Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2024,4,25]]},"reference":[{"key":"2024061000011185100_B1","doi-asserted-by":"crossref","first-page":"589","DOI":"10.1038\/s41580-021-00382-6","article-title":"Molecular mechanisms underlying nucleotide repeat expansion disorders","volume":"22","author":"Malik","year":"2021","journal-title":"Nat. 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