Module 1 - (Patient) induced Pluripotent Stem Cell (iPSC) Differentiation

Human induced pluripotent cells can be generated by any human somatic cell type. Thus human iPSC may represent the most promising in vitro system to mirror potential effects of a drug or therapy regimen on patients. The possibility to use patient cells who are eligible for gene therapy gives the unique opportunity to measure the genotoxicity of a gene therapy regimen in the real target cells a priori to the treatment. This personalized approach takes into account disease and genetic background of each individual patient.