An international research team led by scientists at St. Jude Children’s Research Hospital says it has found a way to use CRISPR gene editing to help fix sickle cell disease and β-thalassemia in blood cells isolated from patients. The study (“A Genome-Editing Strategy to Treat β-Hemoglobinopathies That Recapitulates a Mutation Associated with a Benign Genetic Condition”), which appears online in Nature Medicine, provides proof-of-principle for a new approach to treat common blood disorders by genome editing, according to the investigators.
“Our approach to gene editing is informed by the known benefits of hereditary persistence of fetal hemoglobin,” said Mitchell J. Weiss, M.D., Ph.D., chair of the St. Jude Department of Hematology and one of the study’s lead authors. “It has been known for some time that individuals with genetic mutations that persistently elevate fetal hemoglobin are resistant to the symptoms of sickle cell disease and β-thalassemia, genetic forms of severe anemia that are common in many regions of the world. We have found a way to use CRISPR gene editing to produce similar benefits.” Click for complete story.