Gene Therapy of the ?-Hemoglobinopathies by Lentiviral Transfer of the ?(A(T87Q))-Globin Gene.

ABSTRACT: ?-globin gene disorders are the most prevalent inherited diseases worldwide and result from abnormal ?-globin synthesis or structure. Novel therapeutic approaches are being developed in an effort to move beyond palliative management. Gene therapy, by ex vivo lentiviral transfer of a therapeutic ?-globin gene derivative (?(AT87Q)-globin) to hematopoietic stem cells, driven by cis-regulatory elements that confer high, erythroid-specific expression, has been evaluated in human clinical trials over the past 8 years. ?(AT87Q)-globin is used both as a strong inhibitor of HbS polymerization and as a biomarker. While long-term studies are underway in multiple centers in Europe and in the United States, proof-of-principle of efficacy and safety has already been obtained in multiple patients with ?-thalassemia and sickle cell disease.

SUBMITTER: Negre O 

PROVIDER: S-EPMC4779296 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature