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MONDAY, January 28, 2019 (HealthDay News) – Recent advances in gene therapy could eventually cure sickle cell disease.
The disease is caused by a single mutation of a gene and occurs mainly in people of African descent. In the United States, about 100,000 people have the disease, which causes excruciating pain, stroke, and premature death. The New York Times reported.
Currently, the only treatment is an expensive and risky bone marrow transplant.
In half a dozen clinical trials planned or underway, researchers are testing genetic therapies for sickle cell disease and some patients in these studies are no longer showing signs of the disease.
One of these patients, Brandon Williams, 21, of Chicago, had four strokes at age 18. Her older sister died of the disease. After experimental gene therapy, it no longer exhibits the symptoms of sickle cell disease, The temperature reported.
Despite promising results, it is unclear whether the effects of the treatment will last and it will probably be at least three years before gene therapy for sickle cell disease is approved.
"We are in unknown territory," said Dr. David A. Williams, chief scientist at Boston Children's Hospital. The temperature.
"This would be the first genetic treatment of a common genetic disease," said Dr. Edward Benz, a professor of medicine at Harvard Medical School. The temperature.
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