Gene therapy for hemophilia.
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ABSTRACT:
SUBMITTER: Rogers GL
PROVIDER: S-EPMC4476626 | biostudies-literature | 2015 Jan
REPOSITORIES: biostudies-literature
Publications
Gene therapy for hemophilia.
Rogers Geoffrey L GL Herzog Roland W RW
Frontiers in bioscience (Landmark edition) 20150101 3
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 cl ...[more]
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