CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia
β-Thalassemia is an inherited hematological disorder that results from genetic changes in the β-globin gene, leading to the reduced or absent synthesis of β-globin. For several decades, the only curative treatment option for β-thalassemia has been allogeneic hematopoietic cell transplantation (allo-...
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-02-01
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| Series: | Thalassemia Reports |
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| Online Access: | https://www.mdpi.com/2039-4365/13/1/6 |
| _version_ | 1797608867023028224 |
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| author | Udani Gamage Kesari Warnakulasuriya Sonali Hansika Gayathri N. Silva |
| author_facet | Udani Gamage Kesari Warnakulasuriya Sonali Hansika Gayathri N. Silva |
| author_sort | Udani Gamage |
| collection | DOAJ |
| description | β-Thalassemia is an inherited hematological disorder that results from genetic changes in the β-globin gene, leading to the reduced or absent synthesis of β-globin. For several decades, the only curative treatment option for β-thalassemia has been allogeneic hematopoietic cell transplantation (allo-HCT). Nonetheless, rapid progress in genome modification technologies holds great potential for treating this disease and will soon change the current standard of care for β-thalassemia. For instance, the emergence of the CRISPR/Cas9 genome editing platform has opened the door for precision gene editing and can serve as an effective molecular treatment for a multitude of genetic diseases. Investigational studies were carried out to treat β-thalassemia patients utilizing CRISPR-based CTX001 therapy targeting the fetal hemoglobin silencer BCL11A to restore γ-globin expression in place of deficient β-globin. The results of recently carried out clinical trials provide hope of CTX001 being a promising one-time therapeutic option to treat β-hemoglobinopathies. This review provides an insight into the key scientific steps that led to the development and application of novel CRISPR/Cas9–based gene therapies as a promising therapeutic platform for transfusion-dependent β-thalassemia (TDT). Despite the resulting ethical, moral, and social challenges, CRISPR provides an excellent treatment option against hemoglobin-associated genetic diseases. |
| first_indexed | 2024-03-11T05:49:32Z |
| format | Article |
| id | doaj.art-b7336f71dab64ae1b5742d3a971381a1 |
| institution | Directory Open Access Journal |
| issn | 2039-4365 |
| language | English |
| last_indexed | 2024-03-11T05:49:32Z |
| publishDate | 2023-02-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Thalassemia Reports |
| spelling | doaj.art-b7336f71dab64ae1b5742d3a971381a12023-11-17T14:12:07ZengMDPI AGThalassemia Reports2039-43652023-02-01131516910.3390/thalassrep13010006CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-ThalassemiaUdani Gamage0Kesari Warnakulasuriya1Sonali Hansika2Gayathri N. Silva3Department of Chemistry, University of Colombo, Colombo 00700, Sri LankaDepartment of Chemistry, University of Colombo, Colombo 00700, Sri LankaDepartment of Chemistry, University of Colombo, Colombo 00700, Sri LankaDepartment of Chemistry, University of Colombo, Colombo 00700, Sri Lankaβ-Thalassemia is an inherited hematological disorder that results from genetic changes in the β-globin gene, leading to the reduced or absent synthesis of β-globin. For several decades, the only curative treatment option for β-thalassemia has been allogeneic hematopoietic cell transplantation (allo-HCT). Nonetheless, rapid progress in genome modification technologies holds great potential for treating this disease and will soon change the current standard of care for β-thalassemia. For instance, the emergence of the CRISPR/Cas9 genome editing platform has opened the door for precision gene editing and can serve as an effective molecular treatment for a multitude of genetic diseases. Investigational studies were carried out to treat β-thalassemia patients utilizing CRISPR-based CTX001 therapy targeting the fetal hemoglobin silencer BCL11A to restore γ-globin expression in place of deficient β-globin. The results of recently carried out clinical trials provide hope of CTX001 being a promising one-time therapeutic option to treat β-hemoglobinopathies. This review provides an insight into the key scientific steps that led to the development and application of novel CRISPR/Cas9–based gene therapies as a promising therapeutic platform for transfusion-dependent β-thalassemia (TDT). Despite the resulting ethical, moral, and social challenges, CRISPR provides an excellent treatment option against hemoglobin-associated genetic diseases.https://www.mdpi.com/2039-4365/13/1/6β-thalassemiaβ-globinγ-globinBCL11ACRISPR/Cas9 technologyCTX001 therapy |
| spellingShingle | Udani Gamage Kesari Warnakulasuriya Sonali Hansika Gayathri N. Silva CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia Thalassemia Reports β-thalassemia β-globin γ-globin BCL11A CRISPR/Cas9 technology CTX001 therapy |
| title | CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia |
| title_full | CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia |
| title_fullStr | CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia |
| title_full_unstemmed | CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia |
| title_short | CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia |
| title_sort | crispr gene therapy a promising one time therapeutic approach for transfusion dependent β thalassemia crispr cas9 gene editing for β thalassemia |
| topic | β-thalassemia β-globin γ-globin BCL11A CRISPR/Cas9 technology CTX001 therapy |
| url | https://www.mdpi.com/2039-4365/13/1/6 |
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