Ground state naïve pluripotent stem cells and CRISPR/Cas9 gene correction for β-thalassemia.
作者信息
Finotti Alessia, Borgatti Monica, Gambari Roberto
机构信息
Department of Life Sciences and Biotechnology, Ferrara University, Ferrara, Italy.
Department of Life Sciences and Biotechnology, Ferrara University, Ferrara, Italy; Biotechnology Center, Ferrara University, Ferrara, Italy.
出版信息
Stem Cell Investig. 2016 Oct 25;3:66. doi: 10.21037/sci.2016.09.21. eCollection 2016.
Abstract
摘要
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本文引用的文献
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A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.一种治疗β-珠蛋白生成障碍性贫血的基因组编辑策略，该策略重现了与一种良性遗传疾病相关的突变。
Nat Med. 2016 Sep;22(9):987-90. doi: 10.1038/nm.4170. Epub 2016 Aug 15.
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Combining Single Strand Oligodeoxynucleotides and CRISPR/Cas9 to Correct Gene Mutations in β-Thalassemia-induced Pluripotent Stem Cells.结合单链寡脱氧核苷酸与CRISPR/Cas9校正β-地中海贫血诱导多能干细胞中的基因突变
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Customizing the genome as therapy for the β-hemoglobinopathies.定制基因组作为β-地中海贫血的治疗方法。
Blood. 2016 May 26;127(21):2536-45. doi: 10.1182/blood-2016-01-678128. Epub 2016 Apr 6.
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