Photo Source: NPR
For the first time ever, a human patient with sickle cell disease has been treated with genetically-modified cells that have been edited by CRISPR technology—and new data says that the experimental treatment is working.
Up until now, the genetic blood disorder has been incurable. Sickle cell patients such as Victoria Gray—the patient who underwent the treatment in mid-2019—experience frequent bouts of physical pain and are often forced to get blood transfusions in order to ensure that their body has enough healthy red blood cells to carry oxygen throughout their body.
Since Gray was treated with a CTX001 infusion of edited cells, however, the 34-year-old is already experiencing noticeable health improvements. Not only have clinical followups shown that the treatment is safe, but it has also dramatically reduced the amount of pain Gray experienced as a result of the disease.