In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are
currently in clinical development, gene therapy holds the promise of a lasting cure with a …

In the last decade, enormous progress has been made in the development of gene therapy
for hemophilia A and B. After the first encouraging results of intravenously administered …

Hemophilia is an X-linked inherited bleeding disorder, resulting from defects in the F8
(hemophilia A) or F9 (hemophilia B) genes. Persons with hemophilia have bleeding …

Gene therapy offers the potential for a cure for patients with hemophilia by establishing
continuous endogenous expression of factor VIII or factor IX (FIX) following transfer of a …

The X-linked bleeding disorder hemophilia causes frequent and exaggerated bleeding that
can be life-threatening if untreated. Conventional therapy requires frequent intravenous …

The hemophilias are a group of severe bleeding disorders resulting from the lack of
functional blood coagulation factor VIII (hemophilia A) or factor IX (hemophilia B). Factor IX …

Recent phase I/II adeno-associated viral vector-mediated gene therapy clinical trials have
reported remarkable success in ameliorating disease phenotype in hemophilia A and B …

Hemophilia A and hemophilia B are both X chromosome-linked recessive bleeding
disorders that affect about 1 in 5000 males and result from a deficiency in the coagulation …

Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX
genes. The palliative treatment of choice is based on the use of safe and effective …

The clinical potential of hemophilia gene therapy has now been pursued for the past 30
years, and there is a realistic expectation that this goal will be achieved within the next …