• Hematol. Oncol. Clin. North Am. · Oct 2017

    Review

    Gene Therapy Approaches to Hemoglobinopathies.

    • Giuliana Ferrari, Marina Cavazzana, and Fulvio Mavilio.
    • San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), Istituto Scientifico Ospedale San Raffaele, Via Olgettina 58, Milan 20132, Italy; Vita-Salute San Raffaele University, Milan, Italy.
    • Hematol. Oncol. Clin. North Am. 2017 Oct 1; 31 (5): 835-852.

    AbstractGene therapy for hemoglobinopathies is currently based on transplantation of autologous hematopoietic stem cells genetically modified with a lentiviral vector expressing a globin gene under the control of globin transcriptional regulatory elements. Preclinical and early clinical studies showed the safety and potential efficacy of this therapeutic approach as well as the hurdles still limiting its general application. In addition, for both beta-thalassemia and sickle cell disease, an altered bone marrow microenvironment reduces the efficiency of stem cell harvesting as well as engraftment. These hurdles need be addressed for gene therapy for hemoglobinopathies to become a clinical reality.Copyright © 2017 Elsevier Inc. All rights reserved.

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