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: Javid Gaziev, Guido Lucarelli
• Journal: Hematology/oncology clinics of North America (Hematol Oncol Clin North Am) Vol. 24 Issue 6 Pg. 1145-63 (Dec 2010) ISSN: 1558-1977 [Electronic] United States
• PMID: 21075285 (Publication Type: Journal Article, Review)
• Copyright: Copyright © 2010 Elsevier Inc. All rights reserved.

Topics

• Anemia, Sickle Cell (genetics, therapy)
• Cord Blood Stem Cell Transplantation
• Hematopoietic Stem Cell Transplantation (methods)
• Hemoglobinopathies (genetics, therapy)
• Histocompatibility Testing
• Humans
• Thalassemia (genetics, therapy)
• Tissue Donors
• Transplantation Conditioning (methods)
• Transplantation, Homologous