Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice

• Main Authors: Karine Sii-Felice, Marie Giorgi, Philippe Leboulch, Emmanuel Payen
• Other Authors: Brigham and Women's Hospital
• Format: Article
• Published: 2019
• Subjects: Biochemistry, Genetics and Molecular Biology; Medicine
• Online Access: https://repository.li.mahidol.ac.th/handle/123456789/45101
• Institution: Mahidol University

© 2018 ISEH – Society for Hematology and Stem Cells The β-hemoglobinopathies, transfusion-dependent β-thalassemia and sickle cell disease, are the most prevalent inherited disorders worldwide and affect millions of people. Many of these patients have a shortened life expectancy and suffer from seve...