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Showing 1–2 of 2 results
Advanced filters: Author: Ashlesha Odak Clear advanced filters

  • A gene therapy phase 1 trial in patients with β-thalassemia shows transplantation of autologous CD34+ cells transduced with a lentiviral globin vector after reduced-intensity conditioning achieves long-term engraftment, albeit not transfusion independence, with benign clonal expansions, warranting cautious monitoring of patients.

    • Farid Boulad
    • Aurelio Maggio
    • Michel Sadelain
    Research
    Nature Medicine
    Volume: 28, P: 63-70

  • Introducing chimeric antigen receptors into the endogenous T-cell receptor locus reduces tonic signalling, averts accelerated T-cell differentiation and delays T-cell exhaustion, leading to enhanced function and anti-tumour efficacy compared to random integrations.

    • Justin Eyquem
    • Jorge Mansilla-Soto
    • Michel Sadelain
    Research
    Nature
    Volume: 543, P: 113-117