THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES
In the last ten years, a consistent number of clinical studies have evaluated different gene approaches for the treatment of patients with sickle cell disease (SCD) and transfusion-dependent b-thalassemia (TDT). Initial studies of gene therapy for hemoglobinopathies involved the use of lentiviral v...
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| Format: | Article |
| Language: | English |
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PAGEPress Publications
2024-08-01
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| Series: | Mediterranean Journal of Hematology and Infectious Diseases |
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| Online Access: | https://www.mjhid.org/mjhid/article/view/5768 |
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| author | Ugo Testa Giuseppe Leone Maria Domenica Cappellini |
| author_facet | Ugo Testa Giuseppe Leone Maria Domenica Cappellini |
| author_sort | Ugo Testa |
| collection | DOAJ |
| description |
In the last ten years, a consistent number of clinical studies have evaluated different gene approaches for the treatment of patients with sickle cell disease (SCD) and transfusion-dependent b-thalassemia (TDT). Initial studies of gene therapy for hemoglobinopathies involved the use of lentiviral vectors to add functional copies of the gene encoding b-globin in defective CD34 cells; more recently, gene editing techniques have been used involving either CRISPR-Cas9, transcription activation-like effector protein nuclease, zinc finger nuclease, and base editing to either induce fetal hemoglobin production at therapeutic levels or to genetically repair the underlying molecular defect causing the disease.
Here, we review recent gene editing studies that have started the development of a new era in the treatment of hemoglobinopathies and, in general, monoallelic hereditary diseases.
Keywords: Gene editing; gene therapy; Hemoglobinopathies; Thalassemia; Sickle Cell Anemia.
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| format | Article |
| id | doaj-art-c45c231579d8442a954580e3710636ef |
| institution | Kabale University |
| issn | 2035-3006 |
| language | English |
| publishDate | 2024-08-01 |
| publisher | PAGEPress Publications |
| record_format | Article |
| series | Mediterranean Journal of Hematology and Infectious Diseases |
| spelling | doaj-art-c45c231579d8442a954580e3710636ef2025-02-03T10:21:32ZengPAGEPress PublicationsMediterranean Journal of Hematology and Infectious Diseases2035-30062024-08-0116110.4084/MJHID.2024.068THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIESUgo Testa0Giuseppe LeoneMaria Domenica Cappellini1ISSUniversità di Milano In the last ten years, a consistent number of clinical studies have evaluated different gene approaches for the treatment of patients with sickle cell disease (SCD) and transfusion-dependent b-thalassemia (TDT). Initial studies of gene therapy for hemoglobinopathies involved the use of lentiviral vectors to add functional copies of the gene encoding b-globin in defective CD34 cells; more recently, gene editing techniques have been used involving either CRISPR-Cas9, transcription activation-like effector protein nuclease, zinc finger nuclease, and base editing to either induce fetal hemoglobin production at therapeutic levels or to genetically repair the underlying molecular defect causing the disease. Here, we review recent gene editing studies that have started the development of a new era in the treatment of hemoglobinopathies and, in general, monoallelic hereditary diseases. Keywords: Gene editing; gene therapy; Hemoglobinopathies; Thalassemia; Sickle Cell Anemia. https://www.mjhid.org/mjhid/article/view/5768Gene editing; gene therapy; Hemoglobinopathies; Thalassemia; Sickle Cell Anemia. |
| spellingShingle | Ugo Testa Giuseppe Leone Maria Domenica Cappellini THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES Mediterranean Journal of Hematology and Infectious Diseases Gene editing; gene therapy; Hemoglobinopathies; Thalassemia; Sickle Cell Anemia. |
| title | THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES |
| title_full | THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES |
| title_fullStr | THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES |
| title_full_unstemmed | THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES |
| title_short | THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES |
| title_sort | therapeutic gene editing for hemoglobinopathies |
| topic | Gene editing; gene therapy; Hemoglobinopathies; Thalassemia; Sickle Cell Anemia. |
| url | https://www.mjhid.org/mjhid/article/view/5768 |
| work_keys_str_mv | AT ugotesta therapeuticgeneeditingforhemoglobinopathies AT giuseppeleone therapeuticgeneeditingforhemoglobinopathies AT mariadomenicacappellini therapeuticgeneeditingforhemoglobinopathies |