Millions of people globally, a vast majority of them Africans, suffer from sickle cell disease. The disease also affects people with southern European, Middle Eastern or Asian backgrounds, or those who are Hispanic. "Researchers have worked for decades on improving treatment and finding a cure, but experts say the effort has been hindered by chronic underfunding, in part because most of the estimated 100,000 people in the United States who have the disease are African-American, often poor or of modest means".
The trial, run by Dr. David A. Williams, an expert in the biology of blood-forming stem cells at Boston Children’s, and Dr. Esrick has a straightforward goal:
“We’re going to re-educate the blood cells and make them think they are still in the fetus,” Dr. Williams said.
the first 3 adult patients to go under the trial were cure from Sickle Cell Disease so the F.D.A said Boston Children's Hospital could move to teenagers.
A 16 years old girl was given the gene therapy infusion at Boston Children’s, and her symptoms disappeared after a couple of months. She has her six-month checkup on Dec. 16 and the doctors gave the exciting news that She had no signs of sickle cell disease and her total hemoglobin level was so high it was nearly normal.
To read more of the story click the link below: