Lenti in red: progress in gene therapy for human hemoglobinopathies.
Hemoglobinopathies are caused by abnormal structure or synthesis of hemoglobin chains and represent serious monogenic disorders. A new study demonstrates that lentiviral vectors can express clinically relevant levels of human transgenic beta-globin in red cells of xenografted mice. While some safety concerns must be addressed, this study is an important step toward potential clinical trials of gene therapy for hemoglobinopathies.
von Kalle
Christof
C
Division of Expermental Hematology, Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, Ohio 45229, USA.
Baum
Christopher
C
Williams
David A
DA
D000818:Animals; D015316:Genetic Therapy; D005822:Genetic Vectors; D005914:Globins / Q000235:genetics; D006453:Hemoglobinopathies / Q000235:genetics* / Q000628:therapy*; D006801:Humans; D051379:Mice; D019076:Transgenes
D005914:Globins