make it easier for patients and providers to learn and engage with the research, clinical trials, and other activities happening across the country.
“The engagement of patients will be a cornerstone of this effort,” said Benz. “Patients will work alongside researchers in developing and recruiting for clinical trials.”
Currently, the only cure for sickle cell disease is a bone marrow transplant, a procedure in which a sick patient receives bone marrow from a healthy, genetically-compatible sibling donor. However, transplants are too risky for many adults, and only about 18 percent of children with sickle cell disease have a healthy, matched sibling donor.
The Cure Sickle Cell Initiative seeks to develop cures for a far broader group of individuals with the disease, and it is initially focusing on gene therapies that modify the patient’s own hematopoietic stem cells (HSCs), which make red and other blood cells.
These modified HSCs can then be given back to the patient via a