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CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration
DC Field | Value | Language |
---|---|---|
dc.contributor.author | Koo, Taeyoung | - |
dc.contributor.author | Park, Sung Wook | - |
dc.contributor.author | Jo, Dong Hyun | - |
dc.contributor.author | Kim, Daesik | - |
dc.contributor.author | Kim, Jin Hyoung | - |
dc.contributor.author | Cho, Hee-Yeon | - |
dc.contributor.author | Kim, Jeungeun | - |
dc.contributor.author | Kim, Jeong Hun | - |
dc.contributor.author | Kim, Jin-Soo | - |
dc.date.accessioned | 2020-04-27T12:59:54Z | - |
dc.date.available | 2020-04-27T12:59:54Z | - |
dc.date.created | 2019-07-05 | - |
dc.date.created | 2019-07-05 | - |
dc.date.created | 2019-07-05 | - |
dc.date.created | 2019-07-05 | - |
dc.date.created | 2019-07-05 | - |
dc.date.issued | 2018-12 | - |
dc.identifier.citation | Nature Communications, Vol.9 No.1, p. 1855 | - |
dc.identifier.issn | 2041-1723 | - |
dc.identifier.other | 77667 | - |
dc.identifier.uri | https://hdl.handle.net/10371/165703 | - |
dc.description.abstract | LbCpf1, derived from Lachnospiraceae bacterium ND2006, is a CRISPR RNA-guided endonuclease and holds promise for therapeutic applications. Here we show that LbCpf1 can be used for therapeutic gene editing in a mouse model of age-related macular degeneration (AMD). The intravitreal delivery of LbCpf1, targeted to two angiogenesis-associated genes encoding vascular endothelial growth factor A (Vegfa) and hypoxia inducing factor 1a (Hif1a), using adeno-associated virus, led to efficient gene disruption with no apparent off-target effects in the retina and retinal pigment epithelium (RPE) cells. Importantly, LbCpf1 targeted to Vegfa or Hif1a in RPE cells reduced the area of laser-induced choroidal neovascularization as efficiently as aflibercept, an anti-VEGF drug currently used in the clinic, without inducing cone dysfunction. Unlike aflibercept, LbCpf1 targeted to Vegfa or Hif1a achieved a long-term therapeutic effect on CNV, potentially avoiding repetitive injections. Taken together, these results indicate that LbCpf1-mediated in vivo genome editing to ablate pathologic angiogenesis provides an effective strategy for the treatment of AMD and other neovascularization-associated diseases. | - |
dc.language | 영어 | - |
dc.publisher | Nature Publishing Group | - |
dc.title | CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration | - |
dc.type | Article | - |
dc.contributor.AlternativeAuthor | 김진수 | - |
dc.contributor.AlternativeAuthor | 김정훈 | - |
dc.identifier.doi | 10.1038/s41467-018-04175-y | - |
dc.citation.journaltitle | Nature Communications | - |
dc.identifier.wosid | 000431772000003 | - |
dc.identifier.scopusid | 2-s2.0-85047068898 | - |
dc.citation.number | 1 | - |
dc.citation.startpage | 1855 | - |
dc.citation.volume | 9 | - |
dc.identifier.sci | 000431772000003 | - |
dc.description.isOpenAccess | Y | - |
dc.contributor.affiliatedAuthor | Jo, Dong Hyun | - |
dc.contributor.affiliatedAuthor | Kim, Jeong Hun | - |
dc.contributor.affiliatedAuthor | Kim, Jin-Soo | - |
dc.type.docType | Article | - |
dc.description.journalClass | 1 | - |
dc.subject.keywordPlus | LEBERS CONGENITAL AMAUROSIS | - |
dc.subject.keywordPlus | ENDOTHELIAL GROWTH-FACTOR | - |
dc.subject.keywordPlus | RNA-GUIDED ENDONUCLEASE | - |
dc.subject.keywordPlus | HUMAN-CELLS | - |
dc.subject.keywordPlus | CRISPR-CAS | - |
dc.subject.keywordPlus | DIGENOME-SEQ | - |
dc.subject.keywordPlus | IN-VIVO | - |
dc.subject.keywordPlus | CPF1 | - |
dc.subject.keywordPlus | MICE | - |
dc.subject.keywordPlus | SPECIFICITIES | - |
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