Children with SCID carry genetic defects that prevent their immune systems from working. Without treatment, most die from infection in their first two years of life.
For decades, the treatment has been to get transplants of blood-forming stem cells from the bone marrow of matched siblings or other donors who have healthy immune systems.If you liked this article, please give it a quick review on ycombinator or StumbleUpon. Thanks
Such transplants can effectively cure the disorder. But only about one in five children with SCID have a perfectly matched donor.
Bone marrow from partially matched donors can also be used. But those mismatched transplants are much more risky. About one in three children who have them die from the procedure.
Lessons learned from the SCID trials have spurred studies to find effective gene-based treatments for other blood cell diseases, including sickle cell anemia.
“Twenty years ago, nothing was working," Kohn says. "Ten years ago, these treatments started to work, but with complications. The hope is that the next decade will bring highly effective treatments with few complications.”