The Scientific World Journal

The Scientific World Journal / 2010 / Article
Special Issue

Hemaglobinopathies

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Review Article | Open Access

Volume 10 |Article ID 418163 | https://doi.org/10.1100/tsw.2010.67

Ajay Perumbeti, Punam Malik, "Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New Perspective", The Scientific World Journal, vol. 10, Article ID 418163, 11 pages, 2010. https://doi.org/10.1100/tsw.2010.67

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

Academic Editor: Susan Perrine
Received17 Jun 2009
Revised24 Feb 2010
Accepted19 Mar 2010

Abstract

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.


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