Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation.

ABSTRACT: β-Thalassemia (BT) is one of the most common genetic diseases worldwide and is caused by mutations affecting β-globin production. The only curative treatment is allogenic hematopoietic stem/progenitor cells (HSPCs) transplantation, an approach limited by compatible donor availability and immunological complications. Therefore, transplantation of autologous, genetically-modified HSPCs is an attractive therapeutic option. However, current gene therapy strategies based on the use of lentiviral vectors are not equally effective in all patients and CRISPR/Cas9 nuclease-based strategies raise safety concerns. Thus, base editing strategies aiming to correct the genetic defect in patients' HSPCs could provide safe and effective treatment. Here, we developed a strategy to correct one of the most pr

SUBMITTER: Hardouin G 

PROVIDER: S-EPMC10651780 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature