Success against sickle-cell would be “the first genetic cure of a common genetic disease” and could free tens of thousands of Americans from agonizing pain.
Researchers are trying to address sickle-cell disease at the genetic level, and it’s having drastic results so far among the patients participating in clinical trials, the New York Times reports.
- The experimental gene therapy treatments are still in their early stages, and it could be at least 3 years before one is approved. But a handful of the enrollees no longer show signs of the disease.
- Currently, the only way to treat those with sickle-cell is through a bone marrow transplant, which is dangerous, expensive and uncommon.
The bottom line: “This would be the first genetic cure of a common genetic disease,” Dr. Edward Benz, a professor at Harvard Medical School, told the NYT.