Getting Back to Normal: Correcting SCN by Universal or Precision Strikes.
作者信息
Mehta Hrishikesh M, Corey Seth J
机构信息
Department of Pediatrics and Cancer Biology, Cleveland Clinic, 9500 Euclid Avenue, Cleveland, OH 44195, USA.
Department of Pediatrics and Cancer Biology, Cleveland Clinic, 9500 Euclid Avenue, Cleveland, OH 44195, USA.
出版信息
Mol Ther. 2020 Dec 2;28(12):2525-2526. doi: 10.1016/j.ymthe.2020.11.005. Epub 2020 Nov 10.
Abstract
摘要
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CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia.CRISPR-Cas9 介导的造血干细胞和祖细胞中 ELANE 突变矫正治疗严重先天性中性粒细胞减少症。
Mol Ther. 2020 Dec 2;28(12):2621-2634. doi: 10.1016/j.ymthe.2020.08.004. Epub 2020 Aug 8.
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Inducible expression of a disease-associated mutation impairs granulocytic differentiation, without eliciting an unfolded protein response.诱导表达一种与疾病相关的突变会损害粒细胞分化，而不会引发未折叠蛋白反应。
J Biol Chem. 2020 May 22;295(21):7492-7500. doi: 10.1074/jbc.RA120.012366. Epub 2020 Apr 16.
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CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors.基于 CRISPR 的治疗性基因组编辑：策略和 AAV 载体的体内递送。
Cell. 2020 Apr 2;181(1):136-150. doi: 10.1016/j.cell.2020.03.023.
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Nat Rev Dis Primers. 2017 Jun 8;3:17032. doi: 10.1038/nrdp.2017.32.
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Game of clones: the genomic evolution of severe congenital neutropenia.克隆之博弈：严重先天性中性粒细胞减少症的基因组进化
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