Reprogrammed Crigler-Najjar syndrome cells (University of Cambridge)

Induced pluripotent stem cells (iPSCs) were generated from fibroblasts derived from a 2-month-old Caucasian male with clinical features of Crigler-Najjar syndrome, carrying a 13 base-pair deletion in exon 2 of UGT1A1 gene . The donated tissue was infected with a mix of moloney murine leukemia virus–derived vectors, each containing the coding sequences of one of the following human genes: OCT4, SOX2, c-MYC, and KLF4. Cells express pluripotency markers, demonstrate alkaline phosphatase activity, and form teratomas which contain cells from all the three germ layers.