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In vivo gene editing via homology-independent targeted integration for adrenoleukodystrophy treatment
DC Field | Value | Language |
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dc.contributor.author | 조성래 | - |
dc.contributor.author | 서정화 | - |
dc.date.accessioned | 2021-12-28T16:40:37Z | - |
dc.date.available | 2021-12-28T16:40:37Z | - |
dc.date.issued | 2022-01 | - |
dc.identifier.issn | 1525-0016 | - |
dc.identifier.uri | https://ir.ymlib.yonsei.ac.kr/handle/22282913/186746 | - |
dc.description.abstract | Adrenoleukodystrophy (ALD) is caused by various pathogenic mutations in the X-linked ABCD1 gene, which lead to metabolically abnormal accumulations of very long-chain fatty acids in many organs. However, curative treatment of ALD has not yet been achieved. To treat ALD, we applied two different gene-editing strategies, base editing and homology-independent targeted integration (HITI), in ALD patient-derived fibroblasts. Next, we performed in vivo HITI-mediated gene editing using AAV9 vectors delivered via intravenous administration in the ALD model mice. We found that the ABCD1 mRNA level was significantly increased in HITI-treated mice, and the plasma levels of C24:0-LysoPC (lysophosphatidylcholine) and C26:0-LysoPC, sensitive diagnostic markers for ALD, were significantly reduced. These results suggest that HITI-mediated mutant gene rescue could be a promising therapeutic strategy for human ALD treatment. | - |
dc.description.statementOfResponsibility | restriction | - |
dc.language | English | - |
dc.publisher | Academic Press | - |
dc.relation.isPartOf | MOLECULAR THERAPY | - |
dc.rights | CC BY-NC-ND 2.0 KR | - |
dc.title | In vivo gene editing via homology-independent targeted integration for adrenoleukodystrophy treatment | - |
dc.type | Article | - |
dc.contributor.college | College of Medicine (의과대학) | - |
dc.contributor.department | Dept. of Rehabilitation Medicine (재활의학교실) | - |
dc.contributor.googleauthor | Sung-Ah Hong | - |
dc.contributor.googleauthor | Jung Hwa Seo | - |
dc.contributor.googleauthor | Soohyun Wi | - |
dc.contributor.googleauthor | Eul Sik Jung | - |
dc.contributor.googleauthor | Jihyeon Yu | - |
dc.contributor.googleauthor | Gue-Ho Hwang | - |
dc.contributor.googleauthor | Ji Hea Yu | - |
dc.contributor.googleauthor | Ahreum Baek | - |
dc.contributor.googleauthor | Soeon Park | - |
dc.contributor.googleauthor | Sangsu Bae | - |
dc.contributor.googleauthor | Sung-Rae Cho | - |
dc.contributor.localId | A03831 | - |
dc.relation.journalcode | J02271 | - |
dc.identifier.eissn | 1525-0024 | - |
dc.identifier.url | https://www.sciencedirect.com/science/article/pii/S152500162100304X | - |
dc.subject.keyword | ABCD1 | - |
dc.subject.keyword | CRISPR | - |
dc.subject.keyword | adrenoleukodystrophy | - |
dc.subject.keyword | base editing | - |
dc.subject.keyword | gene therapy | - |
dc.subject.keyword | genome editing | - |
dc.subject.keyword | homology-independent targeted integration | - |
dc.subject.keyword | very long-chain fatty acid | - |
dc.contributor.alternativeName | Cho, Sung Rae | - |
dc.contributor.affiliatedAuthor | 조성래 | - |
dc.citation.volume | 30 | - |
dc.citation.startPage | 119 | - |
dc.citation.endPage | 129 | - |
dc.identifier.bibliographicCitation | MOLECULAR THERAPY, Vol.30 : 119-129, 2022-01 | - |
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