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Reprogramming based gene therapy for inherited red blood cell disorders.

作者信息

Xu Xiuling, Qu Jing, Suzuki Keiichiro, Li Mo, Zhang Weizhou, Liu Guang-Hui, Izpisua Belmonte Juan Carlos

机构信息

National Laboratory of Biomacromolecules, Institute of Biophysics, Chinese Academy of Sciences, Beijing 100101, China.

出版信息

Cell Res. 2012 Jun;22(6):941-4. doi: 10.1038/cr.2012.54. Epub 2012 Apr 3.

DOI:10.1038/cr.2012.54
PMID:22473006
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3367524/
Abstract
摘要

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本文引用的文献

1
Overcoming reprogramming resistance of Fanconi anemia cells.克服范可尼贫血细胞的重编程抵抗。
Blood. 2012 Jun 7;119(23):5449-57. doi: 10.1182/blood-2012-02-408674. Epub 2012 Feb 27.
2
Genetic correction of β-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice.β-地中海贫血症患者特异性 iPS 细胞的基因矫正及其在改善辐照 SCID 小鼠血红蛋白生成中的应用。
Cell Res. 2012 Apr;22(4):637-48. doi: 10.1038/cr.2012.23. Epub 2012 Feb 7.
3
In vitro generation of red blood cells for transfusion: a model for regenerative medicine.用于输血的红细胞的体外生成:再生医学模型。
Regen Med. 2012 Jan;7(1):1-2. doi: 10.2217/rme.11.108.
4
Efficient correction of hemoglobinopathy-causing mutations by homologous recombination in integration-free patient iPSCs.在无整合的患者诱导多能干细胞中通过同源重组有效校正导致血红蛋白病的突变。
Cell Res. 2011 Dec;21(12):1740-4. doi: 10.1038/cr.2011.186. Epub 2011 Nov 22.
5
In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases.利用工程化锌指核酸酶在人诱导多能干细胞中对镰状细胞贫血突变进行原位基因校正。
Stem Cells. 2011 Nov;29(11):1717-26. doi: 10.1002/stem.718.
6
Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease.从一名患有镰状细胞病的成年患者中诱导的人多能干细胞中对一个点突变的基因进行位点特异性修正。
Blood. 2011 Oct 27;118(17):4599-608. doi: 10.1182/blood-2011-02-335554. Epub 2011 Aug 31.
7
Targeted gene correction of laminopathy-associated LMNA mutations in patient-specific iPSCs.靶向纠正患者特异性 iPSC 中与层粘连蛋白病相关的 LMNA 突变。
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8
[Hemolytic anemia in adults: Main causes and diagnostic procedure].[成人溶血性贫血:主要病因及诊断方法]
Presse Med. 2011 May;40(5):470-85. doi: 10.1016/j.lpm.2010.11.013. Epub 2011 Feb 4.
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Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells.基因组安全港可允许地中海贫血诱导多能干细胞中高β-珠蛋白转基因的表达。
Nat Biotechnol. 2011 Jan;29(1):73-8. doi: 10.1038/nbt.1717. Epub 2010 Dec 12.
10
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Nature. 2010 Nov 25;468(7323):521-6. doi: 10.1038/nature09591. Epub 2010 Nov 7.