Induced pluripotent stem cells: a novel frontier in the study of human primary immunodeficiencies.
Induced pluripotent stem cells: a novel frontier in the study of human primary immunodeficiencies.
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DOI：
10.1016/j.jaci.2010.11.008
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发表时间：
2011-06
期刊：
影响因子：
--
通讯作者：
Notarangelo LD
中科院分区：
文献类型：
--
作者：
Pessach IM;Ordovas-Montanes J;Zhang SY;Casanova JL;Giliani S;Gennery AR;Al-Herz W;Manos PD;Schlaeger TM;Park IH;Rucci F;Agarwal S;Mostoslavsky G;Daley GQ;Notarangelo LD
The novel ability to epigenetically reprogram somatic cells into induced pluripotent stem cells through the exogenous expression of transcription promises to revolutionize the study of human diseases. Here we report on the generation of 25 induced pluripotent stem cell lines from 6 patients with various forms of Primary Immunodeficiencies, affecting adaptive and/or innate immunity. Patients’ dermal fibroblasts were reprogrammed by expression of four transcription factors, OCT4, SOX2, KLF4, and c-MYC using a single excisable polycistronic lentiviral vector. Induced pluripotent stem cells derived from patients with primary immunodeficiencies show a stemness profile that is comparable to that observed in human embryonic stem cells. Following in vitro differentiation into embryoid bodies, pluripotency of the patient-derived indiced pluripotent stem cells lines was demonstrated by expression of genes characteristic of each of the three embryonic layers. We have confirmed the patient-specific origin of the induced pluripotent stem cell lines, and ascertained maintenance of karyotypic integrity. By providing a limitless source of diseased stem cells that can be differentiated into various cell types in vitro, the repository of induced pluripotent stem cell lines from patients with primary immunodeficiencies represents a unique resource to investigate the pathophysiology of hematopoietic and extra-hematopoietic manifestations of these diseases, and may assist in the development of novel therapeutic approaches based on gene correction.
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影响因子：
14.2
作者：
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通讯作者：
Orange, Jordan S.
影响因子：
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通讯作者：
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影响因子：
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作者：
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通讯作者：
Studer, Lorenz
DOI：
10.1073/pnas.0910012107
发表时间：
2010-03-02
影响因子：
11.1
作者：
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通讯作者：
Zhang, Su-Chun
影响因子：
5.6
作者：
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通讯作者：
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