Nucleic Acid Therapy for β-Thalassemia

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, IndonesiaCorrespondence: Annette d’Arqom Department of Pharmacolog...

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Autor principal:	d'Arqom A
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2020
Materias:	rnai splice switching oligonucleotides gene editing targeted therapy good health and well-being Medicine (General) R5-920
Acceso en línea:	https://doaj.org/article/12aafec9aea94e45aa9007931c5834ee
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spelling	oai:doaj.org-article:12aafec9aea94e45aa9007931c5834ee2021-12-02T09:18:20ZNucleic Acid Therapy for β-Thalassemia1177-5491https://doaj.org/article/12aafec9aea94e45aa9007931c5834ee2020-09-01T00:00:00Zhttps://www.dovepress.com/nucleic-acid-therapy-for-beta-thalassemia-peer-reviewed-article-BTThttps://doaj.org/toc/1177-5491Annette 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, IndonesiaCorrespondence: Annette d’Arqom Department of Pharmacology and Therapy, Faculty of MedicineUniversitas Airlangga, Jl. Mayjen Prof. Dr. Moestopo 47, Surabaya 60131, East Java, IndonesiaEmail annette-d-a@fk.unair.ac.idAbstract: 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-beingd'Arqom ADove Medical Pressarticlernaisplice switching oligonucleotidesgene editingtargeted therapygood health and well-beingMedicine (General)R5-920ENBiologics: Targets & Therapy, Vol Volume 14, Pp 95-105 (2020)
institution	DOAJ
collection	DOAJ
language	EN
topic	rnai splice switching oligonucleotides gene editing targeted therapy good health and well-being Medicine (General) R5-920
spellingShingle	rnai splice switching oligonucleotides gene editing targeted therapy good health and well-being Medicine (General) R5-920 d'Arqom A Nucleic Acid Therapy for β-Thalassemia
description	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, IndonesiaCorrespondence: Annette d’Arqom Department of Pharmacology and Therapy, Faculty of MedicineUniversitas Airlangga, Jl. Mayjen Prof. Dr. Moestopo 47, Surabaya 60131, East Java, IndonesiaEmail annette-d-a@fk.unair.ac.idAbstract: 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
format	article
author	d'Arqom A
author_facet	d'Arqom A
author_sort	d'Arqom A
title	Nucleic Acid Therapy for β-Thalassemia
title_short	Nucleic Acid Therapy for β-Thalassemia
title_full	Nucleic Acid Therapy for β-Thalassemia
title_fullStr	Nucleic Acid Therapy for β-Thalassemia
title_full_unstemmed	Nucleic Acid Therapy for β-Thalassemia
title_sort	nucleic acid therapy for β-thalassemia
publisher	Dove Medical Press
publishDate	2020
url	https://doaj.org/article/12aafec9aea94e45aa9007931c5834ee
work_keys_str_mv	AT darqoma nucleicacidtherapyforbetathalassemia
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