Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice
DC Field | Value | Language |
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dc.contributor.author | Yu, Wenhan | - |
dc.contributor.author | Mookherjee, Suddhasil | - |
dc.contributor.author | Chaitankar, Vijender | - |
dc.contributor.author | Hiriyanna, Suja | - |
dc.contributor.author | Kim, Jung-Woong | - |
dc.contributor.author | Brooks, Matthew | - |
dc.contributor.author | Ataeijannati, Yasaman | - |
dc.contributor.author | Sun, Xun | - |
dc.contributor.author | Dong, Lijin | - |
dc.contributor.author | Li, Tiansen | - |
dc.contributor.author | Swaroop, Anand | - |
dc.contributor.author | Wu, Zhijian | - |
dc.date.accessioned | 2021-12-24T01:41:25Z | - |
dc.date.available | 2021-12-24T01:41:25Z | - |
dc.date.issued | 2017-03 | - |
dc.identifier.issn | 2041-1723 | - |
dc.identifier.uri | https://scholarworks.bwise.kr/cau/handle/2019.sw.cau/52726 | - |
dc.description.abstract | In retinitis pigmentosa, loss of cone photoreceptors leads to blindness, and preservation of cone function is a major therapeutic goal. However, cone loss is thought to occur as a secondary event resulting from degeneration of rod photoreceptors. Here we report a genome editing approach in which adeno-associated virus (AAV)-mediated CRISPR/Cas9 delivery to postmitotic photoreceptors is used to target the Nrl gene, encoding for Neural retina-specific leucine zipper protein, a rod fate determinant during photoreceptor development. Following Nrl disruption, rods gain partial features of cones and present with improved survival in the presence of mutations in rod-specific genes, consequently preventing secondary cone degeneration. In three different mouse models of retinal degeneration, the treatment substantially improves rod survival and preserves cone function. Our data suggest that CRISPR/Cas9-mediated NRL disruption in rods may be a promising treatment option for patients with retinitis pigmentosa. | - |
dc.language | 영어 | - |
dc.language.iso | ENG | - |
dc.publisher | NATURE PORTFOLIO | - |
dc.title | Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice | - |
dc.type | Article | - |
dc.identifier.doi | 10.1038/ncomms14716 | - |
dc.identifier.bibliographicCitation | NATURE COMMUNICATIONS, v.8 | - |
dc.description.isOpenAccess | Y | - |
dc.identifier.wosid | 000396106200001 | - |
dc.citation.title | NATURE COMMUNICATIONS | - |
dc.citation.volume | 8 | - |
dc.type.docType | Article | - |
dc.publisher.location | 독일 | - |
dc.subject.keywordPlus | ROD PHOTORECEPTORS | - |
dc.subject.keywordPlus | CONE PHOTORECEPTORS | - |
dc.subject.keywordPlus | GENE-THERAPY | - |
dc.subject.keywordPlus | TRANSCRIPTIONAL REGULATION | - |
dc.subject.keywordPlus | MOUSE MODEL | - |
dc.subject.keywordPlus | RAT MODEL | - |
dc.subject.keywordPlus | EXPRESSION | - |
dc.subject.keywordPlus | CRISPR-CAS9 | - |
dc.subject.keywordPlus | MUTATIONS | - |
dc.subject.keywordPlus | RHODOPSIN | - |
dc.relation.journalResearchArea | Science & Technology - Other Topics | - |
dc.relation.journalWebOfScienceCategory | Multidisciplinary Sciences | - |
dc.description.journalRegisteredClass | sci | - |
dc.description.journalRegisteredClass | scie | - |
dc.description.journalRegisteredClass | scopus | - |
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