title: Hemoglobin gene therapy for β-thalassemia.
abstract: Allogeneic stem cell transplantation currently is the only curative option for severe β-thalassemia and sickle cell disease. Human globin gene therapy with autotransplantation of transduced human hematopoietic stem cells is an exciting alternative approach to a potential cure. One patient with thalassemia has recently been reported to have clinical benefit after lentiviral human β-globin gene therapy. He has not required blood transfusions for almost 2 years. Most of the patient's gene correction and new human β-globin gene expression is caused by the expansion of a single clone in which the corrective transgene is inserted into an Hmga2 gene.
authors.affiliation: Columbia University, New York, NY, USA. ab13@columbia.edu
authors.forename: Arthur
authors.initials: A
authors.lastname: Bank
mesh_terms: D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors / Q000235:genetics; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D055544:beta-Globins / Q000235:genetics*; D017086:beta-Thalassemia / Q000235:genetics / Q000628:therapy*
chemical_list: D055544:beta-Globins