Gene therapy for hemophilia

被引：42

作者：

Rogers, Geoffrey L. ^{[1
]}

Herzog, Roland W. ^{[1
]}

机构：

[1] Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL 32610 USA

来源：

FRONTIERS IN BIOSCIENCE-LANDMARK | 2015年 / 20卷

关键词：

Gene therapy; Hemophilia; Factor IX; Factor VIII; AAV; Lentivirus; Review; HUMAN-FACTOR-VIII; COAGULATION-FACTOR-VIII; ADENOASSOCIATED VIRAL VECTORS; SLEEPING-BEAUTY TRANSPOSON; HEMATOPOIETIC STEM-CELLS; LONG-TERM EXPRESSION; HUMAN-FACTOR-IX; PORCINE FACTOR-VIII; HIGH-LEVEL EXPRESSION; SINUSOIDAL ENDOTHELIAL-CELLS;

D O I：

10.2741/4324

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Hemophilia is an X-linked inherited bleeding disorder consisting of two classifications, hemophilia A and hemophilia B, depending on the underlying mutation. Although the disease is currently treatable with intravenous delivery of replacement recombinant clotting factor, this approach represents a significant cost both monetarily and in terms of quality of life. Gene therapy is an attractive alternative approach to the treatment of hemophilia that would ideally provide life-long correction of clotting activity with a single injection. In this review, we will discuss the multitude of approaches that have been explored for the treatment of both hemophilia A and B, including both in vivo and ex vivo approaches with viral and nonviral delivery vectors.

引用

页码：556 / 603

页数：48

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