[HTML][HTML] Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment
GF Pierce, D Lillicrap, SW Pipe… - Journal of Thrombosis …, 2007 - Elsevier
Summary The World Federation of Hemophilia estimates that of the 400 000 individuals
worldwide with hemophilia, 300 000 receive either no, or very sporadic, treatment. Thus,
considerable innovation will be required to provide cost‐effective therapies/cures for all
affected individuals. The high cost of prophylactic regimens hampers their widespread use,
which further justifies the search for novel cost‐effective therapies and ultimately a cure. Five
gene transfer phase I clinical trials have been conducted using either direct in vivo gene …
worldwide with hemophilia, 300 000 receive either no, or very sporadic, treatment. Thus,
considerable innovation will be required to provide cost‐effective therapies/cures for all
affected individuals. The high cost of prophylactic regimens hampers their widespread use,
which further justifies the search for novel cost‐effective therapies and ultimately a cure. Five
gene transfer phase I clinical trials have been conducted using either direct in vivo gene …
