[HTML][HTML] Lenti in red: progress in gene therapy for human hemoglobinopathies
C Von Kalle, C Baum… - The Journal of clinical …, 2004 - Am Soc Clin Investig
C Von Kalle, C Baum, DA Williams
The Journal of clinical investigation, 2004•Am Soc Clin InvestigHemoglobinopathies 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 β-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.
and represent serious monogenic disorders. A new study demonstrates that lentiviral vectors
can express clinically relevant levels of human transgenic β-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.
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 β-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.
The Journal of Clinical Investigation