Gene-targeting of human pluripotent stem cells to study human T lymphocyte development and immune deficiencies

To obtain fundamental insights in normal T cell development and to investigate the applicability of genome-editing for gene therapy, we will manipulate the genome of human hematopoietic and pluripotent stem cells. This will allow a better correlation between genotypes and phenotypes in patients. A RAG2 knock-out model will be used as a reference.