Progress and prospects in stem cell therapy

Abstract

In the past few years, progress being made in stem cell studies has incontestably led to the hope of developing cell replacement based therapy for diseases deficient in effective treatment by conventional ways. The induced pluripotent stem cells (iPSCs) are of great interest of cell therapy research because of their unrestricted self-renewal and differentiation potentials. Proof of principle studies have successfully demonstrated that iPSCs technology would substantially benefit clinical studies in various areas, including neurological disorders, hematologic diseases, cardiac diseases, liver diseases and etc. On top of this, latest advances of gene editing technologies have vigorously endorsed the possibility of obtaining disease-free autologous cells from patient specific iPSCs. Here in this review, we summarize current progress of stem cell therapy research with special enthusiasm in iPSCs studies. In addition, we compare current gene editing technologies and discuss their potential implications in clinic application in the future.

Figure 1

Potential strategy of human PSC-based therapy for human diseases.

Figure 2

Mutant genes in patient-derived iPSCs are corrected by the use of gene editing tools (eg ZFN, TALEN, CRISPR/Cas, HDAdV). The differentiation derivatives from corrected patient iPSCs hold the potential to be employed in autologous cell therapy.