Boston Children’s Hospital
Sickle cell disease affects approximately 100,000 Americans, and it occurs in about 1 out of every 365 African-American births and in about 1 out of every 16,300 Hispanic-American births.
Another estimate reveals that about 1 in 13 African-American babies are born with the sickle cell trait. The disease can cause a wide array of health problems, including the swelling of hands and feet, an increase in the frequency of infections, a delay in growth or puberty, vision problems, and chronic pain, due to bone and joint damage or ulcers.
But what exactly is sickle cell disease? According to the Centers for Disease Control and Prevention, sickle cell disease (or SCD) “is a group of inherited red blood cell disorders. Red blood cells contain hemoglobin, a protein that carries oxygen. Healthy red blood cells are round and move through small blood vessels to carry oxygen to all body parts. In someone with SCD, the hemoglobin is abnormal, which causes the red blood cells to become hard and sticky and look like a C-shaped farm tool called a ‘sickle.’ ”
Why They Made the Worthy 100: Dr. David A. Williams is a renowned hematologist affiliated with Boston Children’s Hospital and the Dana-Farber Cancer Institute. He is one of the leading medical researchers at the forefront of making headway in treating and working to find a cure for sickle cell disease. Williams and his team created a new type of gene therapy, a medical technique that uses genetic material to prevent or cure a disease. There are various approaches to using gene therapy in patients. Still, Williams and his lab relied on a technique to restart a patient’s fetal hemoglobin, which is the dominant form of hemoglobin present in the fetus during gestation. It’s a type of hemoglobin that functions very well in low-oxygen environments (such as a fetus in the womb). It also has anti-sickling characteristics.