Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients.

ABSTRACT: Beta-thalassemia is a common monogenic disorder due to mutations in the beta-globin gene and gene therapy, based on autologous transplantation of genetically corrected haematopoietic stem cells (HSCs), holds the promise to treat patients lacking a compatible bone marrow (BM) donor. We recently showed correction of murine beta-thalassemia by gene transfer in HSCs with the GLOBE lentiviral vector (LV), expressing a transcriptionally regulated human beta-globin gene. Here, we report successful correction of thalassemia major in human cells, by studying a large cohort of pediatric patients of diverse ethnic origin, carriers of different mutations and all candidates to BM transplantation. Extensive characterization of BM-derived CD34(+) cells before and following gene transfer shows the achieve

SUBMITTER: Roselli EA 

PROVIDER: S-EPMC3377331 | biostudies-literature | 2010 Aug

REPOSITORIES: biostudies-literature