Abstract

Gene therapy seems to be moving past the “proof of principle” phase with two new treatments described at the 70th Scientific Sessions of the American Heart Association meeting held in Orlando, Fla November 9 through November 12, 1997. “It is an opening door,” said Valentin Fuster, MD, incoming American Heart Association President and director of the Cardiovascular Institute at Mount Sinai Medical Center in New York City. “It’s not definitive, but it’s certainly very exciting.” In each case, researchers used genes to tinker with or get around disease-causing problems rather than striking directly at the cause of the disorder itself. These kinds of “incremental” gene therapies will probably become more common in the next few years as the field itself matures. The two groups took very separate attacks on the problem of occlusion in limbs, or limb ischemia, which affects between 100 000 and 200 000 people in the United States each year. One was an in vivo experiment injecting genes directly into muscles to encourage the growth of collateral blood vessels. The second was ex vivo and involved bathing a vein graft in a solution containing an oligodeoxynucleotide (ODN) that is a transcription factor decoy to block gene activity. Clinical researchers at St. Elizabeth’s Medical Center in Boston, Mass, attempted to stimulate the growth of collateral blood vessels in the patients’ legs that had been occluded by atherosclerotic lesions. In a technique that Jeffrey Isner, MD, of St. Elizabeth’s and Tufts Medical School called “therapeutic angiogenesis,” the researchers inserted the gene for vascular endothelial growth factor (VEGF) directly into the muscles of the patients’ legs. His hope was that VEGF would encourage the growth of blood vessels that would provide flow to the ischemic areas of the leg. Patients could not be candidates for bypass therapy or other types …

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