Gene therapy having big sickle-cell disease results

Image result for These Patients Had Sickle-Cell Disease. Experimental Therapies Might Have Cured Them.

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.

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