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Gene Therapy For Hemophilia: "Now Within Reach"

EXPERIMENTAL GENE THERAPIES MAY SOON BRING RELIEF TO PATIENTS WITH HEMOPHILIA. On June 1, doctors at the University of Pittsburgh began inserting the gene for blood-clotting factor directly into patients' bodies--the first such clinical trial for this disease. The next week, at University of North Carolina (UNC), researchers reported that a single dose of the blood-clotting gene prompted mice to produce the crucial protein continuously for 11 months. Avigen Inc., a biotech company, also has plans for gene-therapy trials. "This is not pie in the sky anymore," says Dr. Christopher E. Walsh, clinical director of UNC's Gene Therapy Center. A gene therapy treatment for hemophilia "is now within reach."

While blood-clotting genes are well-known, doctors until now lacked an effective delivery mechanism. A standard approach--removing cells from patients' bodies, inserting the desired gene, and returning the cells--has had only limited success with other types of diseases.