Lenti in red: progress in gene therapy for human hemoglobinopathies

被引：12

作者：

von Kalle, C

Baum, C

Williams, DA

机构：

[1] Childrens Hosp, Med Ctr, Div Expt Hematol, Cincinnati, OH 45229 USA

[2] Univ Cincinnati, Coll Med, Cincinnati, OH USA

[3] Hannover Med Sch, Dept Hematol Oncol, Hannover, Germany

来源：

JOURNAL OF CLINICAL INVESTIGATION | 2004年 / 114卷 / 07期

关键词：

D O I：

10.1172/JCI200423132

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

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 (see the related article beginning on page 953). While some safety concerns must be addressed, this study is an important step toward potential clinical trials of gene therapy for hemoglobinopathies.

引用

页码：889 / 891

页数：3

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