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Nucleic Acid Therapy for β-Thalassemia

Authors d'Arqom A

Received 2 June 2020

Accepted for publication 20 August 2020

Published 15 September 2020 Volume 2020:14 Pages 95—105

DOI https://doi.org/10.2147/BTT.S265767

Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 2

Editor who approved publication: Professor Shein-Chung Chow


Annette d’Arqom1,2

1Graduate Program in Molecular Medicine, Faculty of Science, Mahidol University, Bangkok, Thailand; 2Department of Pharmacology and Therapy, Faculty of Medicine, Universitas Airlangga, Surabaya, Indonesia

Correspondence: Annette d’Arqom Department of Pharmacology and Therapy, Faculty of Medicine
Universitas Airlangga, Jl. Mayjen Prof. Dr. Moestopo 47, Surabaya 60131, East Java, Indonesia
Email annette-d-a@fk.unair.ac.id

Abstract: 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.

Keywords: RNAi, splice switching oligonucleotides, gene editing, targeted therapy, good health and well-being

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