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Volume 10, Pages 644-654
Review Article

Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New Perspective

Divisions of Hematology-Oncology and Experimental Hematology/Cancer Biology, Cancer and Blood Institute, Cincinnati Children's Research Foundation, Cincinnati Children's Hospital Medical Center (CCHMC), Cincinnati, OH, USA

Received 17 June 2009; Revised 24 February 2010; Accepted 19 March 2010

Academic Editor: Susan Perrine

Copyright © 2010 Ajay Perumbeti and Punam Malik.


Gene therapy for β-globinopathies, particularly β-thalassemia and sickle cell anemia, holds promise for the future as a definitive corrective approach for these common and debilitating disorders. Correction of the β-globinopathies using lentivirus vectors carrying the β- or γ-globin genes and elements of the locus control region has now been well established in murine models, and an understanding of "what is required to cure these diseases" has been developed in the first decade of the 21st century. A clinical trial using one such vector has been initiated in France with intriguing results, while other trials are under development. Vector improvements to enhance the safety and efficiency of lentivirus vectors are being explored, while new strategies, including homologous recombination in induced pluripotent cells, for correction of sickle cell anemia have shown proof-of-concept in vitro. Here, a review is provided of the current substantial progress in genetic correction of β-globin disorders.