Adenine base editing and prime editing of chemically derived hepatic progenitors rescue genetic liver disease
DC Field | Value | Language |
---|---|---|
dc.contributor.author | Kim, Yohan | - |
dc.contributor.author | Hong, Sung-Ah | - |
dc.contributor.author | Yu, Jihyeon | - |
dc.contributor.author | Eom, Jeongyun | - |
dc.contributor.author | Jang, Kiseok | - |
dc.contributor.author | Yoon, Sangtae | - |
dc.contributor.author | Hong, Da Hee | - |
dc.contributor.author | Seo, Daekwan | - |
dc.contributor.author | Lee, Seu-Na | - |
dc.contributor.author | Woo, Jae-Sung | - |
dc.contributor.author | Jeong, Jaemin | - |
dc.contributor.author | Bae, Sangsu | - |
dc.contributor.author | Choi, Dongho | - |
dc.date.accessioned | 2022-02-21T14:42:22Z | - |
dc.date.available | 2022-02-21T14:42:22Z | - |
dc.date.created | 2022-02-07 | - |
dc.date.issued | 2021-09-02 | - |
dc.identifier.issn | 1934-5909 | - |
dc.identifier.uri | https://scholar.korea.ac.kr/handle/2021.sw.korea/136391 | - |
dc.description.abstract | DNA base editors and prime editing technology enable therapeutic in situ correction of disease-causing alleles. These techniques could have broad applications for ex vivo editing of cells prior to transplantation in a range of diseases, but it is critical that the target population is efficiently modified and engrafts into the host. Chemically derived hepatic progenitors (CdHs) are a multipotent population capable of robust engraftment and hepatocyte differentiation. Here we reprogrammed hepatocytes from a mouse model of hereditary tyrosinemia type 1 (HT1) into expandable CdHs and successfully corrected the disease-causing mutation using both adenine base editors (ABEs) and prime editors (PEs). ABE- and PE-corrected CdHs repopulated the liver with fumarylacetoacetate hydrolase-positive cells and dramatically increased survival of mutant HT1 mice. These results demonstrate the feasibility of precise gene editing in transplantable cell populations for potential treatment of genetic liver disease. | - |
dc.language | English | - |
dc.language.iso | en | - |
dc.publisher | CELL PRESS | - |
dc.subject | HEPATOCYTE-LIKE CELLS | - |
dc.subject | MOUSE MODEL | - |
dc.subject | STEM-CELLS | - |
dc.subject | LONG-TERM | - |
dc.subject | TRANSPLANTATION | - |
dc.subject | GENERATION | - |
dc.subject | DIFFERENTIATION | - |
dc.subject | REGENERATION | - |
dc.subject | FIBROBLASTS | - |
dc.subject | CONVERSION | - |
dc.title | Adenine base editing and prime editing of chemically derived hepatic progenitors rescue genetic liver disease | - |
dc.type | Article | - |
dc.contributor.affiliatedAuthor | Woo, Jae-Sung | - |
dc.identifier.doi | 10.1016/j.stem.2021.04.010 | - |
dc.identifier.scopusid | 2-s2.0-85113727567 | - |
dc.identifier.wosid | 000692518000011 | - |
dc.identifier.bibliographicCitation | CELL STEM CELL, v.28, no.9, pp.1614 - + | - |
dc.relation.isPartOf | CELL STEM CELL | - |
dc.citation.title | CELL STEM CELL | - |
dc.citation.volume | 28 | - |
dc.citation.number | 9 | - |
dc.citation.startPage | 1614 | - |
dc.citation.endPage | + | - |
dc.type.rims | ART | - |
dc.type.docType | Article | - |
dc.description.journalClass | 1 | - |
dc.description.journalRegisteredClass | scie | - |
dc.description.journalRegisteredClass | scopus | - |
dc.relation.journalResearchArea | Cell Biology | - |
dc.relation.journalWebOfScienceCategory | Cell & Tissue Engineering | - |
dc.relation.journalWebOfScienceCategory | Cell Biology | - |
dc.subject.keywordPlus | CONVERSION | - |
dc.subject.keywordPlus | DIFFERENTIATION | - |
dc.subject.keywordPlus | FIBROBLASTS | - |
dc.subject.keywordPlus | GENERATION | - |
dc.subject.keywordPlus | HEPATOCYTE-LIKE CELLS | - |
dc.subject.keywordPlus | LONG-TERM | - |
dc.subject.keywordPlus | MOUSE MODEL | - |
dc.subject.keywordPlus | REGENERATION | - |
dc.subject.keywordPlus | STEM-CELLS | - |
dc.subject.keywordPlus | TRANSPLANTATION | - |
dc.subject.keywordAuthor | adenine base editor | - |
dc.subject.keywordAuthor | chemically derived hepatic progenitor | - |
dc.subject.keywordAuthor | ex vivo gene editing therapy | - |
dc.subject.keywordAuthor | genetic disorder | - |
dc.subject.keywordAuthor | prime editing | - |
dc.subject.keywordAuthor | regenerative medicine | - |
dc.subject.keywordAuthor | reprogramming | - |
dc.subject.keywordAuthor | tyrosinemia type 1 | - |
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