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Author Correction: Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

Archive ouverte | Magnani, A. | CCSD

International audience

Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

Archive ouverte | Magnani, A. | CCSD

International audience. Patients with Wiskott–Aldrich syndrome (WAS) lacking a human leukocyte antigen-matched donor may benefit from gene therapy through the provision of gene-corrected, autologous hematopoietic st...

Gene Therapy in Patients with Transfusion-Dependent beta-Thalassemia

Archive ouverte | Thompson, A. A. | CCSD

International audience. BACKGROUND Donor availability and transplantation-related risks limit the broad use of allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent beta-thalassemia. A...

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