Gene Therapy

For the first time, researchers have used gene therapy to increase light sensitivity and improve vision in patients who were virtually blind, a finding that offers new hope to hundreds of thousands of patients with inherited forms of vision impairment. Although the patients had a rare form of blindness called Leber's congenital amaurosis, researchers believe the approach can ultimately be used for a broad spectrum of disorders, including retinitis pigmentosa and macular degeneration.

The government has suspended a Seattle company's gene therapy study -- and is reviewing the safety of 28 others around the country -- after learning that a patient died this week. The Food and Drug Administration didn't reveal the cause of death or any details about the patient, who had enrolled in a study of gene therapy for advanced arthritis. The agency said it was investigating what role, if any, therapy played in the death, which occurred Tuesday.

A few hours before she died this summer at age 36, Jolee Mohr lay in a Chicago hospital so swollen by internal bleeding and failing kidneys that her husband decided against bringing their 5-year-old daughter to say goodbye. The girl wouldn't have recognized her mother. Robb Mohr couldn't bring himself to watch her die. He spent his wife's last hours talking with her helpless and puzzled doctors. One vowed to get to the bottom of the illness, and there were several clues to go on.

After a year of scant progress in the once-promising field of gene therapy, German scientists said Sunday that they had successfully used the experimental technique to cure two patients with a rare immune disorder. The patients suffered from chronic granulomatous disease, or CGD, an inherited illness that leaves its victims susceptible to life-threatening infections. The disease, which affects about 1,500 people in the U.S., is caused by a defect in a gene needed to destroy harmful bacteria.

ANDREW KILBARGER was 4 years old when his preschool teacher realized something was terribly wrong. He didn't run or jump, and he couldn't pedal a bike. When his parents had him checked by his pediatrician, even the physician was stunned by the diagnosis: Andrew had Duchenne muscular dystrophy, a rare, inherited, degenerative muscle disorder that afflicts 30,000 in the U.S., almost exclusively boys.

TO the shrill whine of a high-speed drill, neurosurgeon Dr. Paul Larson makes two nickel-sized holes in Shirley Cooper's skull. Guided by a computerized MRI map, he plunges a long, thin needle through one hole and deep into the brain -- and empties the syringe. A very special payload trickles into her brain: genes that, if all goes well, will help her control the movement of her muscles.

Among the most promising gene therapy trials are ones to treat various forms of cancer. More than 100 of these are in the works, taking a variety of approaches: using genes to ramp up the immune system or block the action of other, cancer-causing genes, to name a couple. Most of these -- and, indeed, all gene therapy treatments -- are still in the early stages of development. But one therapy could be on the market in the U.S. within the year.

Scientists for the first time have genetically modified tumor-fighting immune cells, allowing patients to rid themselves of an aggressive form of cancer, according to a study released Thursday. The technique, used to cure two patients with advanced melanoma, paves the way for a new approach to fighting cancer by harnessing -- and boosting -- the body's own immune system instead of relying on toxic chemotherapy and radiation treatments to kill out-of-control tumors.

The first studies of human gene therapy for Parkinson's disease have shown that the technique is safe and can reduce symptoms for patients, two groups of researchers have reported. All of the 24 patients who received therapy in the two separate trials received some benefit and none had any significant side effects, researchers reported at neuroscience meetings Tuesday and last week.

A new type of gene therapy that injects a modified version of HIV into special immune cells appears to hinder the AIDS virus' ability to replicate, according to a new study. In the five HIV-infected patients in the study, the amount of virus remained stable or decreased. The number of T cells -- immune cells that fight the virus -- held steady or increased in four of five patients.