Lenti in red: progress in gene therapy for human hemoglobinopathies

AUTOR(ES)

von Kalle, Christof

FONTE

American Society for Clinical Investigation

RESUMO

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.

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