Gene editing of human pluripotent stem cells (iPSCs) to treat inherited platelet disorders
The Institute for Clinical and Experimental Transfusion Medicine (IKET) has long standing expertise in manufacture and application of conventional blood products as well advance therapeutic Medical Products (ATMPS). At our research group protocols have been developed to produce megakaryocytes from non-G-CSF-stimulated blood donors in order to ex vivo human generate platelets. The main goal of ongoing studies is the large scale production of gen-edited platelets, obtained using the CRISP/Cas9 method, to treat patients with inherited platelet disorders. Moreover, several novel in vivo models have been established to preclinical evaluate platelet disorders. Using humanized mouse models the human thrombopoiesis as well as in vivo human platelet survival and function can be analyzed.
GRT projects
Gene Editing: genetically modified iPSCs to treat inherited platelet to better understand inherited platelet disorders and for platelet transfusion
GRT expertise
iPSCs culture for gene editing