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Sustained Phenotypic Correction of Murine Hemophilia A by In Vivo
Gene Therapy
Sheila Connelly,
Julie L. Andrews,
Angela M. Gallo,
Dawn B. Kayda,
Jiahua Qian,
Leon Hoyer,
Michael J. Kadan,
Mario I. Gorziglia,
Bruce C. Trapnell,
Alan McClelland, and
Michael Kaleko
From the Holland Laboratory, American Red Cross, Rockville; and
Genetic Therapy, Inc, Gaithersburg, MD.
Hemophilia A is caused by a deficiency of blood coagulation factor
VIII (FVIII) and has been widely discussed as a candidate for gene
therapy. While the natural canine model of hemophilia A has been
valuable for the development of FVIII pharmaceutical products, the use
of hemophiliac dogs for gene therapy studies has several limitations
such as expense and the long canine generation time. The recent
creation of two strains of FVIII-deficient mice provides the first
small animal model of hemophilia A. Treatment of
hemophiliac mice of both genotypes with potent, human FVIII-encoding adenoviral vectors resulted in expression of biologically active human
FVIII at levels, which declined, but remained above the human
therapeutic range for over 9 months. The duration of expression and
FVIII plasma levels achieved were similar in both hemophiliac mouse
strains. Treated mice readily survived tail clipping with minimal blood
loss, thus showing phenotypic correction of murine hemophilia A by in
vivo gene therapy.
Blood, Vol. 91 No. 9 (May 1), 1998:
pp. 3273-3281
© 1998 by The American Society of Hematology.
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