Françoise Bernaudin

TL;DR: It is shown that, 33 months after lentiviral β-globin gene transfer, an adult patient with severe βE/β0-thalassaemia dependent on monthly transfusions since early childhood has become transfusion independent for the past 21 months.

TL;DR: Interleukin-12 (IL-12) receptor deficiency was found in otherwise healthy individuals with mycobacterial infections, and mature granulomas were seen, surrounded by T cells and centered with epithelioid and multinucleated giant cells, yet reduced IFN-gamma concentrations were found to be secreted by activated natural killer and T cells.

TL;DR: Allogeneic stem-cell transplantation can be curative in young patients with symptomatic sickle cell disease and Kaplan-Meier estimates of survival and event-free survival at four years were 91 percent and 73 percent, respectively.

TL;DR: Results indicate that HLA-identical sibling HSCT after myeloablative conditioning with ATG should be considered as a standard of care for SCD children who are at high risk for stroke.

TL;DR: Data confirm that allogenic bone marrow transplantation establishes normal erythropoiesis and is associated with improved growth and stable CNS imaging and pulmonary function in most patients.