Therapy for beta-globinopathies: a brief review and determinants for successful and safe correction.
Gene therapy for beta-globinopathies, particularly beta-thalassemia and sickle cell anemia, hold much promise for the future, as a one time cure for these common and debilitating disorders. Correction of the beta-globinopathies using lentivirus vectors (LV) carrying the beta- or gamma-globin genes and elements of the locus control region has been well established in murine models, and a good idea of "what it will take to cure these diseases" has been developed in the first decade of the twenty-first century. A clinical trial using one such vector has been initiated in France while other trials are in development. Vector improvements to enhance the safety and efficiency of LV are being explored, while newer strategies, like homologous recombination in induced pluripotent cells for correction of sickle cell anemia, has been shown as a proof-of-concept. Here we provide a review of current progress in genetic correction of beta-globin disorders.
Perumbeti
Ajay
A
Hematology-Oncology, Cancer and Blood Institute, Cincinnati Children's Research Foundation, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Malik
Punam
P
D000818:Animals; D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000235:genetics / Q000627:therapeutic use; D006678:HIV / Q000235:genetics; D006453:Hemoglobinopathies / Q000235:genetics / Q000628:therapy*; D006801:Humans; D019901:Locus Control Region; D055544:beta-Globins / Q000235:genetics
D055544:beta-Globins