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Efficient correction of hemoglobinopathy-causing mutations by homologous recombination in integration-free patient iPSCs.

作者信息

Li Mo, Suzuki Keiichiro, Qu Jing, Saini Preeti, Dubova Ilir, Yi Fei, Lee Jungmin, Sancho-Martinez Ignacio, Liu Guang-Hui, Izpisua Belmonte Juan Carlos

出版信息

Cell Res. 2011 Dec;21(12):1740-4. doi: 10.1038/cr.2011.186. Epub 2011 Nov 22.

DOI:10.1038/cr.2011.186
PMID:22105484
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3357996/
Abstract
摘要

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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.
2
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.
3
Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection.通过体外筛选揭示锌指核酸酶的脱靶切割特异性。
Nat Methods. 2011 Aug 7;8(9):765-70. doi: 10.1038/nmeth.1670.
4
An unbiased genome-wide analysis of zinc-finger nuclease specificity.锌指核酸酶特异性的无偏基因组分析。
Nat Biotechnol. 2011 Aug 7;29(9):816-23. doi: 10.1038/nbt.1948.
5
Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations.生成仅在两个早发性帕金森病点突变处存在差异的同基因多能干细胞。
Cell. 2011 Jul 22;146(2):318-31. doi: 10.1016/j.cell.2011.06.019. Epub 2011 Jul 14.
6
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。
Cell Stem Cell. 2011 Jun 3;8(6):688-94. doi: 10.1016/j.stem.2011.04.019. Epub 2011 May 19.
7
Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy.从一名回旋性萎缩症患者诱导多能干细胞中进行基因矫正和分析。
Proc Natl Acad Sci U S A. 2011 Apr 19;108(16):6537-42. doi: 10.1073/pnas.1103388108. Epub 2011 Apr 4.
8
A more efficient method to generate integration-free human iPS cells.一种更有效的生成无整合的人诱导多能干细胞的方法。
Nat Methods. 2011 May;8(5):409-12. doi: 10.1038/nmeth.1591. Epub 2011 Apr 3.
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Reprogramming of mouse and human somatic cells by high-performance engineered factors.高性能工程因子对小鼠和人体细胞的重编程。
EMBO Rep. 2011 Apr;12(4):373-8. doi: 10.1038/embor.2011.11. Epub 2011 Mar 11.
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Major challenges for gene therapy of thalassemia and sickle cell disease.地中海贫血症和镰状细胞病的基因治疗的主要挑战。
Curr Gene Ther. 2010 Oct;10(5):404-12. doi: 10.2174/156652310793180724.