Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects.

ABSTRACT: The beta-thalassemias are inherited anemias caused by mutations that severely reduce or abolish expression of the beta-globin gene. Like sickle cell disease, a related beta-globin gene disorder, they are ideal candidates for performing a genetic correction in patient hematopoietic stem cells (HSCs). The most advanced approach utilizes complex lentiviral vectors encoding the human β-globin gene, as first reported by May et al. in 2000. Considerable progress toward the clinical implementation of this approach has been made in the past five years, based on effective CD34+ cell mobilization and improved lentiviral vector manufacturing. Four trials have been initiated in the United States and Europe. Of 16 evaluable subjects, 6 have achieved transfusion independence. One of them developed a dur

SUBMITTER: Mansilla-Soto J 

PROVIDER: S-EPMC4994056 | biostudies-literature | 2016 Apr

REPOSITORIES: biostudies-literature