Search

Induced pluripotent stem cells (iPSCs) have become a new phenomenon of regenerative medicine. It is obvious that they share some common characteristics with embryonic stem cells (ESCs) such as stemness potential, self-renewal p., differentiation p. iPSCs retain their epigenetic memory, allowing becoming patient-specific and so it is not necessary to apply immunosuppressants. The use of ESCs is controversial, because their acquisition is associated with embryo destruction. As a cell source for iPSCs derivation we can use any somatic cells, however, fibroblasts are preferably used due to their easy availability. With transcriptional reprogramming cocktail (OCT4, SOX2, KLF4, c- MYC / OCT4, SOX2, NANOG, LIN28) we can obtain required iPSCs line, which is then further differentiated into neural precursors (NPCs). These cells can be grafted into lesion site, where they can facilitate regeneration by several mechanisms (cell replacement, protective effect, facilitation the expression of trophic factors). Nevertheless, here we are still dealing with the risk of tumorogenesis or low cell derivation efficiency that limits the use of iPSCs in clinical practice. In this thesis we will therefore mainly focus on the therapeutic potential of iPSCs in preclinical studies, their use in the treatment of...

Induced pluripotent stem cells (iPSC) are one of the key discoveries in cell biology of the last decade. These cells are pluripotent stem cells derived from differentiated somatic cells while having used only four exogenous transcription factors. Pluripotent cells, which can be derived from somatic cells carrying genetic mutation, have a great potential to be used in the testing of new drugs and in discovering molecular mechanisms of genetic disorders. iPSC derived from healthy cells can be used in regenerative medicine. Originally, retroviral vectors were used for delivering reprogramming transcription factors to cells. However such approach is not safe for medicinal use, because of the ability of retroviruses to integrate into the host genome. This fact initiated development of safer delivering methods of transcription factors into the cells. In this work I present the overview of methods which have been used for reprogramming including the most common techniques used to test pluripotency. In addition, I will describe iPSC application options for therapy of genetically determined hematological disorders (sickle cell anemia, β-thalassemia, X-linked chronic granulomatous disease) and for modelling of their molecular mechanism (polycythemia vera). Key words: iPSC, reprogramming, hematological...