Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New Perspective
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 elemen...
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| Language: | English |
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Wiley
2010-01-01
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| Series: | The Scientific World Journal |
| Online Access: | http://dx.doi.org/10.1100/tsw.2010.67 |
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| author | Ajay Perumbeti Punam Malik |
| author_facet | Ajay Perumbeti Punam Malik |
| author_sort | Ajay Perumbeti |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-aa721e3b8fec4b4282087b3c8b2645c2 |
| institution | Kabale University |
| issn | 1537-744X |
| language | English |
| publishDate | 2010-01-01 |
| publisher | Wiley |
| record_format | Article |
| series | The Scientific World Journal |
| spelling | doaj-art-aa721e3b8fec4b4282087b3c8b2645c22025-02-03T01:22:24ZengWileyThe Scientific World Journal1537-744X2010-01-011064465410.1100/tsw.2010.67Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New PerspectiveAjay Perumbeti0Punam Malik1Divisions 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, USADivisions 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, USAGene 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.http://dx.doi.org/10.1100/tsw.2010.67 |
| spellingShingle | Ajay Perumbeti Punam Malik Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New Perspective The Scientific World Journal |
| title | Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New Perspective |
| title_full | Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New Perspective |
| title_fullStr | Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New Perspective |
| title_full_unstemmed | Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New Perspective |
| title_short | Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New Perspective |
| title_sort | genetic correction of sickle cell anemia and β thalassemia progress and new perspective |
| url | http://dx.doi.org/10.1100/tsw.2010.67 |
| work_keys_str_mv | AT ajayperumbeti geneticcorrectionofsicklecellanemiaandbthalassemiaprogressandnewperspective AT punammalik geneticcorrectionofsicklecellanemiaandbthalassemiaprogressandnewperspective |