Christopher Vakulskas, director of Enzyme Evolution at Integrated DNA Technologies explains how the company’s novel gene editing tools may one day provide single-treatment cures for diseases caused by single gene mutations, including sickle cell disease.
DHA
“My 12-year-old daughter has sickle thalassemia disease. I as a parent understand how it feels to see your child go through rough times. At first it was so frustrating and overwhelming. She's been in and out the hospital since she was a year old. She's had a lot of blood transfusions, pain, and crises, and at age 5 had a splenectomy… She's so much stronger than I am. I admire her for that.”1
In the US, the Centers for Disease Control and Prevention (CDC) and other medical websites chronicle heart-breaking stories of patients with sickle cell disease (SCD) who experience chronic pain, anaemia, repeated infections, and recurrent hospital stays. The chief symptom of SCD is pain—in fact, sickle cell crises are also referred to as pain crises and are the most common cause for hospitalisation, which may span days or months, and recur throughout life.