Ontology highlight
ABSTRACT:
SUBMITTER: d'Arqom A
PROVIDER: S-EPMC7501986 | biostudies-literature | 2020
REPOSITORIES: biostudies-literature
Biologics : targets & therapy 20200915
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 app ...[more]