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2015 ; 20
(3
): 556-603
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Gene therapy for hemophilia
#MMPMID25553466
Rogers GL
; Herzog RW
Front Biosci (Landmark Ed)
2015[Jan]; 20
(3
): 556-603
PMID25553466
show 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.