期刊
FRONTIERS IN BIOSCIENCE-LANDMARK
卷 20, 期 -, 页码 556-603出版社
FRONTIERS IN BIOSCIENCE INC
DOI: 10.2741/4324
关键词
Gene therapy; Hemophilia; Factor IX; Factor VIII; AAV; Lentivirus; Review
资金
- NIH [P01 HL078810, R01 HL097088, R01 AI51390, R01 HL109442]
- University of Florida College of Medicine
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.
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