The assistance for patients having sickle cell disease might soon come from gene editing to repair the mutation that leads to the disease and augment the patient’s defensive fetal hemoglobin. The latest research shows that by using CRISPR-Cas9 and a remedial short DNA template to fix the sickle cell mutation in an individual’s HSCs (hematopoietic stem cells) could be safe and efficient. Bioengineer Gang Bao—from RU’s (Rice University) Brown School of Engineering—carried the research in partnership with Baylor College of Medicine, Texas Children’s Hospital, and Stanford University. The study was published in the Oxford Academic’s journal Nucleic Acids Research.
Reportedly, sickle cell disease affects around 100,000 Americans and millions other across the globe and is a painful, often deadly inherited condition. The single alteration in hemoglobin subunit beta—also known as beta-globin—forces normal, disc-shaped red blood cells to harden and take attribute “sickle” shapes. These cells can impair vessel walls and form clotting in small blood vessels, blocking the delivery of oxygen to tissues. Now, some people affected with the disease are healed with stem cells from a related, matched donor, which is an option scientists’ believe is available to lesser than 15% of patients. An improved strategy will be changing the patient’s HSPCs (hematopoietic stem and progenitor cells), an option supposedly available to every patient as there will be no menace of rejection.
Speaking of the sickle cell disease, recently, a study showed that athletes suffering from sickle cell characteristics are at more peril to collapse. The study was conducted by scientists from the USF (University of South Florida) and identified a genetic variation known to impact sickle cell disease symptomology. This outcome might explain why some college football players having sickle cell trait encounter adverse clinical results during extreme physical exertion. The study was published in the Southern Medical Journal.