Gene Therapy Makes a Comeback
Maligned for many years after a 1999 study led to the death of an 18-year-old patient, gene therapy may have finally recovered its promise as breakthrough medicine. In recent years the field has taken positive steps, culminating most recently in the successful—and safe—treatment of hemophilia B.
The new treatment uses a modified cold virus, outfitted with a gene that hemophiliacs are missing. The gene in question allows the body to produce Factor IX, which helps the blood to clot.
The study, published online in December by The New England Journal of Medicine, only enrolled six patients—but after the therapy, four of them could halt their regular Factor IX infusions, and the other two could safely reduce the frequency of their infusions.
Coupled with another success earlier this year (treating children born with severe combined immune deficiency), along with new ways of delivering missing genes, the field is offering renewed hope for treating genetic disorders.—Katherine Harmon
Image of gene therapy courtesy of the Genetics Home Reference a service of the U.S. National Library of Medicine®
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