Progress toward the clinical application of autologous induced pluripotent stem cells and gene repair therapy for treatment of familial hypercholesterolemia

Study shows, for the first time, the successful reprogramming of diseased human hepatocytes into induced pluripotent stem cells (iPSC).1

Results also found differentiation into mature hepatocytes was more efficient than that with fibroblast-derived iPSCs.

The generation of diseased hepatocyte-derived human iPSC lines provides a good basis for the study of liver disease pathogenesis.

Such technology could give a potentially unlimited reservoir of cells for the treatment of human liver diseases: generating genetically corrected liver cells via auto-transplantation of genetically modified hepatocytes, thus avoiding liver transplant and lifelong immunosuppression.

### References 1 Bosman, A. et al. Progress toward the clinical application of autologus induced pluripotent stem cells and gene repair therapy for treatment of familial hypercholesterolemia. Abstract presented at The International Liver CongressTM 2011. http://www1.easl.eu/easl2011/program/Orals/187.htm