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May 25, 1994

Treatment for Hemophilia: Gene Therapy vs Transplantation-Reply

Author Affiliations

University of North Carolina Chapel Hill

JAMA. 1994;271(20):1576. doi:10.1001/jama.1994.03510440035023

In Reply.  —Drs Fung and Lo appropriately call attention to transplantation of organs or tissues, including embryonic and fetal tissues that may express antihemophilic proteins, as a way that may lead to a cure for the hemophilias. It has been nearly 27 years since organ transplantation was first used for gene therapy in hemophilia.1 Transplantation of the spleen resulted in elevation of plasma factor VIII levels sufficient to change the phenotype from severe to mild in a canine model of hemophilia A. Liver transplantation was more effective, normalizing or nearly so the factor VIII levels. Over the next 20 years, several patients, as well as hemophilic dogs, were cured of both hemophilia A and hemophilia B by this mode of gene therapy using the wild-type hemophilia genes.With the advent of hepatic cell transplantation, we have examined the feasibility of this mode of allogenic liver cell transfer in the

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