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Gene Therapy Approaches to Hemoglobinopathies

2017; Elsevier BV; Volume: 31; Issue: 5 Linguagem: Inglês

10.1016/j.hoc.2017.06.010

1558-1977

Giuliana Ferrari, Marina Cavazzana, Fulvio Mavilio,

Prenatal Screening and Diagnostics

Gene 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.