Project Title: Targeting of the altered interactions between hematopoietic stem cells and the bone marrow niche in beta-thalassemia.
Project Title: Dissection of immune disregulation in RAG deficiency and development of novel gene correction strategies.
Project Title: Increase safety and efficacy of cancer adoptive immunotherapy
Project Title: Generation of human neuronal models of Dravet Syndrome
Project Title: Harnessing tumor microenvironment by genetic engineering
Project Title: "Gene correction of CD4oLG gene in Tcells and HSPC for the treatment of X-linked HIGM1"