Nucleic Acid Therapy for ?-Thalassemia.

ABSTRACT: ?-thalassemia is caused by mutations in the ?-globin gene which diminishes or abolishes ?-globin chain production. This reduction causes an imbalance of the ?/?-globin chain ratio and contributes to the pathogenesis of the disease. Several approaches to reduce the imbalance of the ?/? 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 ?-globin, either by reducing the ?-globin chain, restoring ?-globin expression and reactivating ?-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 ?-thalassemia.

SUBMITTER: d'Arqom A 

PROVIDER: S-EPMC7501986 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature