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Science Translational Medicine, titled “Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates.”
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CRISPR Editing of Stem Cell Subset Reactivates Fetal Hemoglobin for Treating Genetic Blood Disorders
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Gene editing using CRISPR-Cas9 offers the potential of targeted treatment for a variety of genetic diseases. These include inherited abnormalities of β hemoglobin, which can be indirectly targeted by increasing the amount of healthy fetal hemoglobin even without fully correcting the disease-causing mutation. Humbert et al. published paper in Science Translational Medicine, titled “Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates.”