Ontology highlight
ABSTRACT:
SUBMITTER: Ronen K
PROVIDER: S-EPMC3129560 | biostudies-literature | 2011 Jul
REPOSITORIES: biostudies-literature
Ronen Keshet K Negre Olivier O Roth Shannah S Colomb Charlotte C Malani Nirav N Denaro Maria M Brady Troy T Fusil Floriane F Gillet-Legrand Beatrix B Hehir Kathleen K Beuzard Yves Y Leboulch Philippe P Down Julian D JD Payen Emmanuel E Bushman Frederic D FD
Molecular therapy : the journal of the American Society of Gene Therapy 20110308 7
A lentiviral vector encoding β-globin flanked by insulator elements has been used to treat β-thalassemia (β-Thal) successfully in one human subject. However, a clonal expansion was observed after integration in the HMGA2 locus, raising the question of how commonly lentiviral integration would be associated with possible insertional activation. Here, we report correcting β-Thal in a murine model using the same vector and a busulfan-conditioning regimen, allowing us to investigate efficacy and clo ...[more]