Gene Therapy Shows Promise Against Sickle Cell Disease

Sickle cell trait is an inherited blood disorder that affects 1 million to 3 million Americans with 8 to 10 percent of them being Black. The disease is caused by a single mutation in one gene and mainly occurs in people of African descent which causes agonizing pain, strokes and early death. However, recent advances in gene therapy may eventually lead to a cure for sickle cell disease.

Currently, the only treatment is a risky and costly bone marrow transplant. In a half-dozen clinical trials planned or underway, researchers are testing genetic therapies for sickle cell disease and some patients in those studies no longer have signs of the disease.

One of those patients is 21-year-old Brandon Williams of Chicago, who had four strokes by age 18. His older sister died of the disease. After experimental gene therapy, he no longer has symptoms of sickle cell disease, The New York Times reported.

According to researchers at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center a fetus and newborn produce a different kind of