Construction of thalassemic mouse induced pluripotent stem cells for disease modeling

Induced pluripotent stem cells (iPSCs) have a great potential for disease modeling and therapeutic applications. One of the most common genetic diseases found in South East Asia is thalassemia. β-Thalassemia is an inherited single gene disorder caused by reduction or absence of β-globin chain production. The purpose of this study is to generate mouse iPSCs with β-thalassemia background. Mouse embryonic fibroblast (MEF) cells were obtained from transgenic β-thalassemic mouse, transduced with lentiviral vector carrying 4 transcription factors, Oct4, Klf4, Sox2 and c-Myc (OKSM), and cultured on a feeder layer. The embryonic stem (ES)-like colonies were manually picked up and expanded. The generated mouse iPSCs were similar to mouse embryonic stem cells (mESCs) in morphology, alkaline phosphatase activity and pattern of pluripotent gene expression and able to form teratoma containing tissues derived from all three germ layers. Therefore, we reported here an establishment of β-thalassemic mouse iPSCs which might be used for further disease modeling and therapeutic applications.