Integration-free erythroblast-derived human induced pluripotent stem cells (iPSCs) from an individual with Ataxia-Telangiectasia (A-T)

Niraj Bhatt; Rajib Ghosh; Sanchita Roy; Yongxing Gao; Mary Armanios; Linzhao Cheng; Sonia Franco

doi:10.1016/j.scr.2016.08.003

Integration-free erythroblast-derived human induced pluripotent stem cells (iPSCs) from an individual with Ataxia-Telangiectasia (A-T)

Niraj Bhatt, Rajib Ghosh, Sanchita Roy, Yongxing Gao, Mary Armanios, Linzhao Cheng, Sonia Franco

School of Medicine

Research output: Contribution to journal › Article › peer-review

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Abstract

Peripheral blood was obtained from a 12-year old male carrying bialleleic inactivating mutations at the ATM locus, causing Ataxia-Telangiectasia (A-T). Blood erythroid cells were briefly expanded in vitro and induced pluripotent stem cells (iPSCs) were generated via transfection with episomal vectors carrying hOCT4, hSOX2, hKLF4, hMYC and hBCL2L1. SF-003 iPSCs were free of genomically integrated reprogramming genes, had the specific compound heterozygous mutations, stable karyotype, expressed pluripotency markers and formed teratomas in immunodeficient (NOD scid gamma; NGS) mice. The SF-003 iPSC line may be a useful resource for in vitro modeling of A-T.

Original language	English (US)
Pages (from-to)	205-207
Number of pages	3
Journal	Stem Cell Research
Volume	17
Issue number	2
DOIs	https://doi.org/10.1016/j.scr.2016.08.003
State	Published - Sep 2016

ASJC Scopus subject areas

Developmental Biology
Cell Biology

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title = "Integration-free erythroblast-derived human induced pluripotent stem cells (iPSCs) from an individual with Ataxia-Telangiectasia (A-T)",

abstract = "Peripheral blood was obtained from a 12-year old male carrying bialleleic inactivating mutations at the ATM locus, causing Ataxia-Telangiectasia (A-T). Blood erythroid cells were briefly expanded in vitro and induced pluripotent stem cells (iPSCs) were generated via transfection with episomal vectors carrying hOCT4, hSOX2, hKLF4, hMYC and hBCL2L1. SF-003 iPSCs were free of genomically integrated reprogramming genes, had the specific compound heterozygous mutations, stable karyotype, expressed pluripotency markers and formed teratomas in immunodeficient (NOD scid gamma; NGS) mice. The SF-003 iPSC line may be a useful resource for in vitro modeling of A-T.",

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AU - Bhatt, Niraj

AU - Ghosh, Rajib

AU - Roy, Sanchita

AU - Gao, Yongxing

AU - Armanios, Mary

AU - Cheng, Linzhao

AU - Franco, Sonia

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Integration-free erythroblast-derived human induced pluripotent stem cells (iPSCs) from an individual with Ataxia-Telangiectasia (A-T)

Abstract

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