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C4476626!4476626!25553466
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suck abstract from ncbi

pmid25553466      Front+Biosci+(Landmark+Ed) 2015 ; 20 (ä): 556-603
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  • Gene therapy for hemophilia #MMPMID25553466
  • Rogers GL; Herzog RW
  • Front Biosci (Landmark Ed) 2015[]; 20 (ä): 556-603 PMID25553466show ga
  • 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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