Dear Colleagues,

Human pluripotent stem cells, with a major potential in advanced medical applications, became the center of current cell and molecular biology studies. The fantasy of both scientists and lay people has been captured by culturing immortal human cell lines with a full potential of generating any kind of tissues or organs, including non-regenerating neuronal or heart muscle tissues. The ethical concerns regarding research on human embryonic stem cells disappeared when it was discovered that human induced pluripotent stem cells (iPSCs) can be generated from a range of differentiated cell types, and these iPSCs fully mimic the regenerative and differentiation properties of the embryonic stem cells.

The major current challenges of working with human pluripotent stem cells are to establish proper methods for targeted genetic modulation together with directed tissue differentiation, to devise human cell-based disease models for applications in drug screening, and to develop safe and efficient medical use of pluripotent cell-derived tissues or organs. For all these aims, the specific use and further development of the tools of genetic engineering are essential and remain a challenge. In addition, any stem cell derived preparations for advanced drug screening or therapeutic applications have to comply with the strict regulatory requirements of GLP and GMP environments.

The aim of this Special Issue is to publish original research as well as comprehensive reviews on the recent advances in the field of targeted genetic modifications in human pluripotent stem cells and their derivatives. A special emphasis is placed on the most advanced site-directed gene knock-out, modification and insertion technologies, especially with relevance for the application of human tissues in drug development, advanced diagnostics and cellular therapies.

Balázs Sarkadi
Guest Editor