快猫短视频

Gene therapy back on track

THE black cloud hanging over gene therapy since the death of Jesse Gelsinger
in a trial last year is starting to break up. In the latest of a string of
successes for gene therapy, researchers in Massachusetts have managed to restore
the blood supply to damaged heart muscles.

鈥淭his is solid evidence that gene therapy can have a major, positive impact
on human biology,鈥 says Jeffery Isner, a cardiologist at Tufts University School
of Medicine in Boston, who led the study.

In April, French scientists reported success in treating two infants
suffering from a rare inherited immune disorder sometimes known as bubble boy
disease. And earlier this month, researchers in Texas announced impressive
results from combining gene therapy with chemotherapy to treat cancer
(快猫短视频, 5 August, p 19).

The latest trial involved 13 people with severe chest pain, or angina, that
conventional treatments couldn鈥檛 help. Isner鈥檚 team first mapped areas of the
patients鈥 heart muscles that had no blood supply and appeared dead. Each patient
was then given four injections of the gene for vascular endothelial growth
factor into their heart muscle. Animal tests have shown that VEGF stimulates the
growth of blood vessels.

The treatment had a profound effect. Blood flow to the stricken muscle was
partly restored in four patients and completely restored in five. Most patients
could lead a much more vigorous life. The average number of angina attacks for
all patients dropped from 48 per week to 2, while use of the angina drug
nitroglycerin fell from 55 tablets per week to 2. The findings appear in the
current Circulation.

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