title: Allogeneic cellular gene therapy for hemoglobinopathies.
abstract: Hematopoietic stem cell transplantation (HSCT) offers potentially curative therapy for patients with thalassemia major and sickle cell disease (SCD). Current myeloablative treatment protocols allow the cure of 78% to 90% of patients with thalassemia and 72% to 96% with SCD, depending on disease status at the time of transplantation. The major limitation to successful transplantation is the lack of a suitable HLA-matched family donor. Unrelated donor HSCT is now extensively used to treat thalassemia, with results similar to those obtained following transplantation using HLA-matched sibling donors. Patients who lack a matched related or unrelated donor can now benefit from successful transplantation using haploidentical donors.
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinico Tor Vergata, Viale Oxford 81, Rome, Italy. j.gaziev@fondazioneime.org
authors.forename: Javid
authors.initials: J
authors.lastname: Gaziev
authors.forename: Guido
authors.initials: G
authors.lastname: Lucarelli
mesh_terms: D000755:Anemia, Sickle Cell / Q000235:genetics / Q000628:therapy; D036101:Cord Blood Stem Cell Transplantation; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006453:Hemoglobinopathies / Q000235:genetics / Q000628:therapy*; D006650:Histocompatibility Testing; D006801:Humans; D013789:Thalassemia / Q000235:genetics / Q000628:therapy; D014019:Tissue Donors; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous