Adv Exp Med Biol . 2020;1237:17-28. doi: 10.1007/5584_2019_439.

Gene Editing in Human Pluripotent Stem Cells: Recent Advances for Clinical Therapies

Hatice Burcu Şişli  ¹ , Taha Bartu Hayal  ¹ , Selin Seçkin  ¹ , Selinay Şenkal  ¹ , Binnur Kıratlı  ¹ , Fikrettin Şahin  ¹ , Ayşegül Doğan  ²

Abstract

The identification of human embryonic stem cells and reprogramming technology to obtain induced pluripotent stem cells from adult somatic cells have provided unique opportunity to create human disease models, gene editing strategies and cell therapy options.Development of pluripotent stem cells from somatic cells and genomic manipulation tools enabled to use site specific nucleases in the cell therapy research. Identification of efficient gene manipulation, safe differentiation and use will provide a novel strategy to treat many diseases in the near future. Current available registered clinical trials clearly indicate the need for pluripotent stem cell and gene therapy treatment options. Although gene editing based pluripotent stem cell research is a popular field for research worldwide, improvement of clinical approaches for treatment still remains to be investigated. In this review, we summarized the current situation of gene editing based pluripotent cell therapy developments and applications in clinics.