Publication: Nucleic acid therapy for β-thalassemia
Issued Date
2020-01-01
Resource Type
ISSN
11775491
11775475
11775475
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2-s2.0-85092465066
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Mahidol University
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SCOPUS
Bibliographic Citation
Biologics: Targets and Therapy. Vol.14, (2020), 95-105
Suggested Citation
Annette D’Arqom Nucleic acid therapy for β-thalassemia. Biologics: Targets and Therapy. Vol.14, (2020), 95-105. doi:10.2147/BTT.S265767 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/60111
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Title
Nucleic acid therapy for β-thalassemia
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Abstract
© 2020 d’Arqom. β-thalassemia is caused by mutations in the β-globin gene which diminishes or abolishes β-globin chain production. This reduction causes an imbalance of the α/β-globin chain ratio and contributes to the pathogenesis of the disease. Several approaches to reduce the imbalance of the α/β ratio using several nucleic acid-based technologies such as RNAi, lentiviral mediated gene therapy, splice switching oligonucleotides (SSOs) and gene editing technology have been investigated extensively. These approaches aim to reduce excess free α-globin, either by reducing the α-globin chain, restoring β-globin expression and reactivating γ-globin expression, leading a reduced disease severity, treatment necessity, treatment interval, and disease complications, thus, increasing the life quality of the patients and alleviating economic burden. Therefore, nucleic acid-based therapy might become a potential targeted therapy for β-thalassemia.