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Gene Therapy for Hemoglobinopathies

11 Sept 2018

Abstract excerpt

Gene therapy for β-thalassemia and sickle-cell disease is based on transplantation of genetically corrected, autologous hematopoietic stem cells. Preclinical and clinical studies have shown the safety and efficacy of this therapeutic approach, currently based on lentiviral vectors to transfer a β-globin gene under the transcriptional control of regulatory elements of the β-globin locus. Nevertheless, a number of...

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PMID: 30200783
DOI: 10.1089/hum.2018.122

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