Gene therapy for hemoglobinopathies: progress and challenges
نویسندگان
چکیده
منابع مشابه
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 ...
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ژورنال
عنوان ژورنال: Translational Research
سال: 2013
ISSN: 1931-5244
DOI: 10.1016/j.trsl.2012.12.011