Summary:
After decades of investigation, gene therapy is receiving regulatory
approval to treat hemophilia. However, since gene therapy investigations
were initially conceived, other avenues of treatment have revolutionized
the care of hemophilia. Emergent data is showing that gene therapy may
not be as beneficial as hoped and more toxic than planned. At a minimum,
a reassessment of risk/benefit estimate of gene therapy for hemophilia
is needed.
Emergent data influences
the risk/benefit assessment of hemophilia gene therapy using recombinant
adeno-associated virus