Nucleic acid therapy for β-thalassemia

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7 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)95-105
Number of pages11
JournalBiologics: Targets and Therapy
Volume14
DOIs
Publication statusPublished - 2020

Keywords

  • Gene editing
  • Good health and well-being
  • RNAi
  • Splice switching oligonucleotides
  • Targeted therapy

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