The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia
β-thalassemia is characterized by reduced or absence of β-globin production, resulting in anemia. Current therapies include blood transfusion combined with iron chelation. BM transplantation, although curative, is restricted by the matched donor limitation. Gene therapy, on the other hand, is promis...
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| Format: | Article |
| Language: | English |
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Wiley
2011-01-01
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| Series: | Stem Cells International |
| Online Access: | http://dx.doi.org/10.4061/2011/987980 |
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| author | Ekati Drakopoulou Eleni Papanikolaou Nicholas P. Anagnou |
| author_facet | Ekati Drakopoulou Eleni Papanikolaou Nicholas P. Anagnou |
| author_sort | Ekati Drakopoulou |
| collection | DOAJ |
| description | β-thalassemia is characterized by reduced or absence of β-globin production, resulting in anemia. Current therapies include blood transfusion combined with iron chelation. BM transplantation, although curative, is restricted by the matched donor limitation. Gene therapy, on the other hand, is promising, and its success lies primarily on designing efficient globin vectors that can effectively and stably transduce HSCs. The major breakthrough in β-thalassemia gene therapy occurred a decade ago with the development of globin LVs. Since then, researchers focused on designing efficient and safe vectors, which can successfully deliver the therapeutic transgene, demonstrating no insertional mutagenesis. Furthermore, as human HSCs have intrinsic barriers to HIV-1 infection, attention is drawn towards their ex vivo manipulation, aiming to achieve higher yield of genetically modified HSCs. This paper presents the current status of gene therapy for β-thalassemia, its success and limitations, and the novel promising strategies available involving the therapeutic role of HSCs. |
| format | Article |
| id | doaj-art-234a980524c2424ea39a80c350803504 |
| institution | Kabale University |
| issn | 1687-966X 1687-9678 |
| language | English |
| publishDate | 2011-01-01 |
| publisher | Wiley |
| record_format | Article |
| series | Stem Cells International |
| spelling | doaj-art-234a980524c2424ea39a80c3508035042025-02-03T01:20:44ZengWileyStem Cells International1687-966X1687-96782011-01-01201110.4061/2011/987980987980The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-ThalassemiaEkati Drakopoulou0Eleni Papanikolaou1Nicholas P. Anagnou2Laboratory of Cell and Gene Therapy, Centre for Basic Research, Biomedical Research Foundation of the Academy of Athens (BRFAA), 115 27 Athens, GreeceLaboratory of Cell and Gene Therapy, Centre for Basic Research, Biomedical Research Foundation of the Academy of Athens (BRFAA), 115 27 Athens, GreeceLaboratory of Cell and Gene Therapy, Centre for Basic Research, Biomedical Research Foundation of the Academy of Athens (BRFAA), 115 27 Athens, Greeceβ-thalassemia is characterized by reduced or absence of β-globin production, resulting in anemia. Current therapies include blood transfusion combined with iron chelation. BM transplantation, although curative, is restricted by the matched donor limitation. Gene therapy, on the other hand, is promising, and its success lies primarily on designing efficient globin vectors that can effectively and stably transduce HSCs. The major breakthrough in β-thalassemia gene therapy occurred a decade ago with the development of globin LVs. Since then, researchers focused on designing efficient and safe vectors, which can successfully deliver the therapeutic transgene, demonstrating no insertional mutagenesis. Furthermore, as human HSCs have intrinsic barriers to HIV-1 infection, attention is drawn towards their ex vivo manipulation, aiming to achieve higher yield of genetically modified HSCs. This paper presents the current status of gene therapy for β-thalassemia, its success and limitations, and the novel promising strategies available involving the therapeutic role of HSCs.http://dx.doi.org/10.4061/2011/987980 |
| spellingShingle | Ekati Drakopoulou Eleni Papanikolaou Nicholas P. Anagnou The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia Stem Cells International |
| title | The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia |
| title_full | The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia |
| title_fullStr | The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia |
| title_full_unstemmed | The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia |
| title_short | The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia |
| title_sort | ongoing challenge of hematopoietic stem cell based gene therapy for β thalassemia |
| url | http://dx.doi.org/10.4061/2011/987980 |
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