Supported projects

T201901 – Monitoring of the gene therapy efficiency and study of the immunological reconstitution of patients with immune deficits by determining the copy number into the cells

The Laboratory of Cell and Gene Therapy (LTCG), Department of Biotherapy at Necker-Enfants malades Hospital has expertise in immuno-hematology and innovative therapies. The LTCG supports clinical trials of cell and gene therapy for inherited diseases by targeting hematopoietic cells. Gene therapy is a therapeutic strategy that involves the entry of genes into the cells of a patient to treat a pathology. In order to measure the therapeutic impact of the gene therapy, we develop a precise and absolute quantification of the gene copy number integrated into these cells, and those from the smallest quantities.