Scientists may have cracked the cure for Sickle Cell Disease (SCD) using an editing gene to fix mutated genes. For this study, they used the cell of patients and injected CRISPR-Cas9 to repair mutations in the hematopoietic stem cells. The repaired cells produced healthy hemoglobin.
Jacob Corn from UC Berkeley explained that they are very excited about their findings. He said that even though there are a lot of things that needs to be done before this can be clinically used, but their hopeful that this will help sickle disease patients.
The effects of CRISPR-Cas9 lasted for four months after transplant, Science Daily reported. Mark Walters, one of the researchers, said that their study showed advancement that will be beneficial to patients plagued with the disease.
SCD is a hereditary disorder that can cause hemoglobin abnormality. It is common among African Americans and other African population.
The site of NIH explained SCD is not contagious and most children don't feel the pain caused by the disease. However, they feel chronic pain during their adolescence and adulthood life.
This disease cause hemoglobin to stick together that causes a sickle shape deformation on red blood cells. The cells then get stuck in blood cells causing a blockage that causes the body pain, anemia and organ failure. The scientists believe that their study can help other patients with β-thalassemia, Severe Combined Immunodeficiency (SCID), Wiskott-Aldrich syndrome, Fanconi anemia and HIV infection.
Corn explained that the Sickle Cell Disease is just one of the blood disorders that can be cured by these kind of gene editing. He also stressed that Sickle Cell Disease is just one of the disorders that can cause single genome mutations.
Dana Caroll of University of Utah said that there is a clear path for developing therapies for blood diseases. She explained that it is wonderful to see gene editing technology on practical use.