Hemophilia as trailblazer for gene therapy.

Research output: Contribution to journalEditorial


Hemophilia A and B are inherited bleeding disorders caused by
deficiencies in coagulation factors VIII or IX in the blood
plasma. The drawbacks of the classical protein substitution therapy
fueled interest in alternative treatments by gene therapy.
Hemophilia has been recognized as an ideal target disease for
gene therapy because a relatively modest increase in clotting factor
levels can result in a significant therapeutic benefit. Consequently,
introducing a functional FVIII or FIX gene copy into
the appropriate target cells could ultimately provide a cure for
hemophilic patients. Several cell types have been explored for
hemophilia gene therapy, including hepatocytes, muscle, endothelial
and hematopoietic cells. Both nonviral and viral vectors have
been considered for the development of hemophilia gene therapy,
including transposons, c-retroviral, lentiviral, adenoviral and
adeno-associated viral vectors. Several of these strategies have
resulted in stable correction of the bleeding diathesis in hemophilia
A and B murine as well as canine models, paving the way
towards clinical trials. Although clotting factor expression has
been detected in hemophilic patients treated by gene therapy, the
challenge now lies in obtaining prolonged therapeutic FVIII or
FIX levels in these patients.
Original languageEnglish
Pages (from-to)63-63
Number of pages1
JournalFEBS J
Issue numberJuly
Publication statusPublished - 2011


  • Hemophilia A
  • Hemophilia B
  • Gene therapy


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