首页> Molecular therapy. Methods & clinical development> CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.

CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.

导出 LinkOut

翻译打印收藏纠错

第一作者： Giulia,Bortolussi

第一单位： International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.

作者： Giulia,Bortolussi ^[1] ; Alessandra,Iaconcig ^[1] ; Giulia,Canarutto ^[1] ; Fabiola,Porro ^[1] ; Filippo,Ferrucci ^[1] ; Claudia,Galletta ^[1] ; Cristian,Díaz-Muñoz ^[1] ; Vipin,Rawat ^[1] ; Alessia,De Caneva ^[1] ; Olayemi Joseph,Olajide ^[1] ; Lorena,Zentilin ^[1] ; Silvano,Piazza ^[1] ; Luka,Bočkor ^[1] ; Andrés Fernando,Muro ^[1]

作者单位： International Centre for Genetic Engineering and Biotechnology, Trieste, Italy. ^[1]

关键词 CRISPR-SaCas9 Crigler-Najjar syndrome type I IEM adeno-associated viral vector, AAV bilirubin gene editing inborn error of metabolism liver-directed gene editing neonatal mice neonatal mortality

DOI 10.1016/j.omtm.2023.101161

PMID 38094199

发布时间 2023-12-14

翻译满意度评价：

准确一般字义偏差语意偏离译文作用小提交

浏览0

Molecular therapy. Methods & clinical development

2023年31卷

101161页

相似文献

中文期刊
外文期刊
学位论文
会议论文

浏览0

点击下载本期目录封面

Molecular therapy. Methods & clinical development

2023年31卷

101161页

检索历史清除

CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.

Molecular therapy. Methods & clinical development

相似文献

Molecular therapy. Methods & clinical development

相关期刊

参考文献：
指定格式：	NoteExpress EndNote RefWorks NoteFirst

检索历史 清除

CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.

Molecular therapy. Methods & clinical development

相似文献

Molecular therapy. Methods & clinical development

相关期刊

检索历史清除