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Dr. Mark DeWitt, a leading industry expert, shares his secrets on success with the development of a CRISPR/Cas9-based strategy

Correction of the Sickle Cell Disease Mutation with CRISPR/Cas9 Description Sickle Cell Disease (SCD), one of the world’s most common genetic disorders, causes anemia and progressive multiorgan damage that typically shortens lifespan by decades; currently there is no broadly applicable curative therapy. A universal curative therapy for SCD would address a critical unmet medical need [....]