Gene and cell therapy for beta-thalassemia and sickle cell disease with induced pluripotent stem cells (iPSCs): the next frontier

Haema 2016; 7(1): 100-110

by Eirini P. Papapetrou

Departments of Oncological Sciences, Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA

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Abstract

In recent years, breakthroughs in human pluripotent stem cell (hPSC) research, namely cellular reprogramming and the emergence of sophisticated genetic engineering technologies, have opened new frontiers for cell and gene therapy. The prospect of using hPSCs, either autologous or histocompatible, as targets of genetic modification and their differentiated progeny as cell products for transplantation, presents a new paradigm of regenerative medicine of potential tremendous value for the treatment of blood disorders, including SCD and BT. Despite advances at a remarkable pace and great promise, many roadblocks remain before clinical translation can be realistically considered. Here we discuss the theoretical advantages of cell therapies utilizing hPSC derivatives, recent proof-of-principle studies and the main challenges towards realizing the potential of hPSC therapies in the clinic.