• Marina Cavazzana, Chiara Antoniani, and Annarita Miccio.
• Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, 75015 Paris, France; Biotherapy Clinical Investigation Center, Assistance Publique-Hôpitaux de Paris, INSERM, Groupe Hospitalier Universitaire Ouest, 75015 Paris, France; INSERM UMR 1163, Laboratory of Human Lymphohematopoiesis, 75015 Paris, France; Paris Descartes, Sorbonne Paris Cité University, Imagine Institute, 75015 Paris, France. Electronic address: m.cavazzana@aphp.fr.
• Mol. Ther. 2017 May 3; 25 (5): 1142-1154.

Abstractβ-Thalassemia and sickle cell disease (SCD) are the world's two most widely disseminated hereditary hemoglobinopathies. β-Thalassemia originated in the Mediterranean, Middle Eastern, and Asian regions, and SCD originated in central Africa. However, subsequent population migration means that these two diseases are now global and thus constitute a growing health problem in many countries. Despite remarkable improvements in medical care for patients with β-hemoglobinopathies, there is still only one definitive treatment option: allogeneic hematopoietic stem cell (HSC) transplantation. The development of gene therapy for β-hemoglobinopathies has been justified by (1) the limited availability of human leukocyte antigen (HLA)-identical donors, (2) the narrow window of application of HSC transplantation to the youngest patients, and (3) recent advances in HSC-based gene therapy. The huge ongoing efforts in translational medicine and the high number of related publications show that gene therapy has the potential to become the treatment of choice for patients who lack either an HLA genoidentical sibling or an alternative, medically acceptable donor. In this dynamic scientific context, we first summarize the main steps toward clinical translation of this therapeutic approach and then discuss novel lentiviral- and genome editing-based treatment strategies for β-hemoglobinopathies.Copyright © 2017 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved.

42 keywords...

hide…