Researchers from Johns Hopkins University, the University of Texas Southwestern Medical Center, St. Jude Children's Research Hospital, and Harvard University have developed nanoparticles that can send gene treatment directly to various types of cells in bone marrow to correct mutations that cause sickle cell disease. The researchers used CRISPR/Cas and base gene-editing techniques in a mouse model of sickle cell disease to activate a form of hemoglobin and correct the sickle cell mutation.
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