Week of June 9, 2001; Vol. 159, No. 23
Genetically altered cells ease hemophilia
A gene therapy using skin cells that are genetically modified to make clotting
proteins, multiplied in a lab, and reinjected into a person eases some bleeding
in patients with severe hemophilia.
Roth, D.A., ... and R.F. Selden. 2001. Nonviral transfer of the gene encoding
coagulation factor VIII in patients with severe hemophilia. New England Journal
of Medicine 344(June 7):1735.
Kay, M.A., ... and K.A. High. 2000. Evidence for gene transfer and expression
of factor IX in haemophilia B patients treated with an AAV vector. Nature
Mannucci, P.M., and E.G.D. Tuddenha. 2001. The hemophilias—from royal genes
to gene therapy. New England Journal of Medicine 344(June 7):1773.
Miller, D.G. 2001. Gene therapy for hemophilia. New England Journal of Medicine
Seppa, N. 2000. New gene therapy techniques show potential. Science News
Simpson, S. 1999. Gene injections stem clotting disorder. Science News 155(Jan.
Vermylen, J. 1998. Haemophilia and other inherited bleeding disorders. New
England Journal of Medicine 338(March 5):692.
Further information about hemophilia can be found at
Katherine A. High
Abramson Research Center
3400 Civic Center Boulevard
Philadelphia, PA 19104
Carol K. Kasper
University of Southern California
2400 South Flower Street
Los Angeles, CA 90007
Richard F. Selden
195 Albany Street
Cambridge, MA 02139