Supported projects

T202002 – Optimizing Hematopoietic Stem and Progenitor Cells (HSPC) genetic engineering in inflammatory context

Scientific leader
Dr. Emmanuelle Six

Introducing a therapeutic gene into hematopoietic stem and progenitor cells (HSPCs) is an effective strategy for restoring the functionality of immune cells. However, optimal reconstitution is hampered by the alteration of HSPCs in certain pathological contexts such as inflammation and impact of ex vivo cell culture. This is particularly the case for gene therapy trials involving chronic granulomatosis disease (CGD), characterized by chronic inflammation and in which we have observed great variability in the rate of long-term transplantation of corrected cells.The goal of this project is to optimize the conditions of ex vivo cell culture, by testing different transduction adjuvants, in order to improve the rate of gene correction and HSPCs long-term engraftment in the context of inflammatory pathologies such as CGD, sickle cell disease or acquired immune deficiency syndrome.





CIC-Biothérapie 1416

Institut Imagine

24 Boulevard du Montparnasse

75 015 Paris