Centre d'Investigation Clinique Biothérapies Necker

Recent papers from Centre d'Investigation Clinique Biothérapies Necker

1. Hypomorphic RAG deficiency: impact of disease burden on survival and thymic recovery argues for early diagnosis and HSCTBlood · 2022
2. T-Cell Progenitors As A New Immunotherapy to Bypass Hurdles of Allogeneic Hematopoietic Stem Cell TransplantationFrontiers in Immunology · 2022
3. Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndromeNature Medicine · 2022
4. Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCIDMolecular Therapy — Methods & Clinical Development · 2020
5. Extensive multilineage analysis in patients with mixed chimerism after allogeneic transplantation for sickle cell disease: insight into hematopoiesis and engraftment thresholds for gene therapyHaematologica · 2019
6. Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathiesBlood · 2019
7. A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem CellsMolecular Therapy — Methods & Clinical Development · 2018
8. Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusionHaematologica · 2018
9. Cryptosporidium spp. Infection in Solid Organ TransplantationTransplantation · 2016