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

详细信息    查看全文

• 作者：Niraj Bhatt^a ; ¹ ; Rajib Ghosh^a ; ¹ ; Sanchita Roy^a ; ¹ ; Yongxing Gao^b ; Mary Armanios^c ; Linzhao Cheng^b ; Sonia Franco^a ; ^{sfranco2@jhmi.edu" class="auth_mail" title="E-mail the corresponding author}
• 刊名：Stem Cell Research
• 出版年：2016
• 出版时间：September 2016
• 年：2016
• 卷：17
• 期：2
• 页码：205-207
• 全文大小：468 K

文摘

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.