
CRISPR gene editing reveals new therapeutic approach for blood disorders. Credit: St. Jude Children’s Research Hospital
An international team has found a way to use CRISPR gene editing to help fix sickle cell disease and beta-thalassemia in blood cells isolated from patients. The study in Nature Medicine, provides proof-of-principle for a new approach to treat common blood disorders by genome editing.
“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...
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