Publications by authors named "J-A Ribeil"
N Engl J Med
February 2022
Article Synopsis
- Sickle cell disease causes painful vaso-occlusive events, and researchers are exploring gene therapy using LentiGlobin, which involves modifying patients' hematopoietic stem cells to produce a beneficial form of hemoglobin, HbA.
- A phase 1-2 study was conducted on patients with severe sickle cell disease, focusing on 35 individuals in Group C who experienced frequent vaso-occlusive events, evaluating the treatment's safety and effectiveness after infusion.
- Results showed that all patients in Group C successfully engaged with LentiGlobin, leading to significant improvements in hemoglobin levels and a complete resolution of severe vaso-occlusive events for those evaluated, showcasing the potential of this gene therapy.
Article Synopsis
- The study aims to assess the safety and effectiveness of gene therapy as an alternative to long-term red-cell transfusions for patients with transfusion-dependent β-thalassemia, focusing on the use of a lentiviral vector to modify patients' cells.
- In two phase 1-2 trials involving 22 patients aged 12 to 35, researchers collected and modified the patients' own blood cells with a gene that encodes for adult hemoglobin, followed by reinfusing these cells after conditioning treatment.
- Results showed significant improvements: 12 out of 13 patients with a non-β/β genotype stopped needing transfusions, while those with the β/β genotype experienced a
Article Synopsis
- Gene therapy faces challenges due to complex cellular issues and the need to effectively curb hemoglobin S polymerization.
- A patient treated with a lentiviral vector to add a healthy β-globin gene showed lasting treatment benefits, maintaining high levels of antisickling β-globin without sickle crises for 15 months post-treatment.
