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本文引用的文献

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Methodologies for Improving HDR Efficiency.提高高剂量率效率的方法。
Front Genet. 2019 Jan 7;9:691. doi: 10.3389/fgene.2018.00691. eCollection 2018.
2
Short-hairpin RNA against aberrant mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells.针对异常信使核糖核酸的短发夹RNA在一种新型细胞模型中恢复了β-珠蛋白水平，并在原代造血干细胞中作为β-地中海贫血的单一疗法和联合疗法发挥作用。
Haematologica. 2018 Sep;103(9):e419-e423. doi: 10.3324/haematol.2018.189357. Epub 2018 Apr 26.
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Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies.用于血红蛋白病基因和细胞治疗的珠蛋白链的快速灵敏评估
Hum Gene Ther Methods. 2018 Feb;29(1):60-74. doi: 10.1089/hgtb.2017.190.
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Ensembl 2018.Ensembl 2018.
Nucleic Acids Res. 2018 Jan 4;46(D1):D754-D761. doi: 10.1093/nar/gkx1098.
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Genetic effects on gene expression across human tissues.基因对人体各组织基因表达的影响。
Nature. 2017 Oct 11;550(7675):204-213. doi: 10.1038/nature24277.
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Molecular basis of β thalassemia and potential therapeutic targets.β地中海贫血的分子基础及潜在治疗靶点
Blood Cells Mol Dis. 2018 May;70:54-65. doi: 10.1016/j.bcmd.2017.06.001. Epub 2017 Jun 20.
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Long-Term Engraftment and Fetal Globin Induction upon Gene Editing in Bone-Marrow-Derived CD34 Hematopoietic Stem and Progenitor Cells.骨髓来源的CD34造血干细胞和祖细胞基因编辑后的长期植入及胎儿血红蛋白诱导
Mol Ther Methods Clin Dev. 2017 Jan 11;4:137-148. doi: 10.1016/j.omtm.2016.12.009. eCollection 2017 Mar 17.
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Suitability of small diagnostic peripheral-blood samples for cell-therapy studies.小型诊断性外周血样本在细胞治疗研究中的适用性。
Cytotherapy. 2017 Feb;19(2):311-326. doi: 10.1016/j.jcyt.2016.11.007.
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CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes.CRISPR/Cas9介导的人类三原核受精卵基因编辑。
Protein Cell. 2015 May;6(5):363-372. doi: 10.1007/s13238-015-0153-5. Epub 2015 Apr 18.
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Improved specificity of TALE-based genome editing using an expanded RVD repertoire.利用扩展的 RVD 库提高基于 TALE 的基因组编辑的特异性。
Nat Methods. 2015 May;12(5):465-71. doi: 10.1038/nmeth.3330. Epub 2015 Mar 23.

Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells.

作者信息

Patsali Petros, Turchiano Giandomenico, Papasavva Panayiota, Romito Marianna, Loucari Constantinos C, Stephanou Coralea, Christou Soteroulla, Sitarou Maria, Mussolino Claudio, Cornu Tatjana I, Antoniou Michael N, Lederer Carsten W, Cathomen Toni, Kleanthous Marina

机构信息

Department of Molecular Genetics Thalassemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.

Department of Medical and Molecular Genetics, King's College London, London, UK.

出版信息

Haematologica. 2019 Nov;104(11):e497-e501. doi: 10.3324/haematol.2018.215178. Epub 2019 Apr 19.

DOI:10.3324/haematol.2018.215178
PMID:31004018
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6821606/
Abstract
摘要