Genetic correction of hemophilia A and B was long considered amenable to the available
gene transfer technologies. This assumption has come to fruition with the recent results
of a phase I/II trial for hemophilia B. Here we review the clinical application of
gene therapy for the hemophilia's as a paradigm of the evolution of gene transfer
science and technology. This review is not intended as comprehensive but rather to
highlight current clinical developments of gene therapy for the hemophilias.
Abbreviations:
AAV (adeno-associated virus), BP (branch point), FIX (factor IX), hFIX (human factor IX), PP (polypyrimidine tract), PTM (pre-trans-splicing molecule)To read this article in full you will need to make a payment
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Article info
Publication history
Published online: January 25, 2013
Accepted:
December 28,
2012
Received in revised form:
December 13,
2012
Received:
October 25,
2012
Footnotes
Conflict of interest: None.
Identification
Copyright
© 2013 Mosby, Inc. Published by Elsevier Inc. All rights reserved.
