A team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR - Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of affected patients. (sciencedaily.com)
This inheritance pattern, as well as the equal distribution of the disease in males and females, indicated that there was a single gene responsible for the disease and that it was autosomal (i.e. that the gene was not on the X chromosome). (optigen.com)
Although a team led by Mahajan identified the gene responsible for the disease in 2012, a cure has yet to be developed. (sciencedaily.com)