Highly Efficient Editing of the Beta-Globin Gene in Patient Derived Hematopoietic Stem and Progenitor Cells to Treat Sickle Cell Disease
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DOI: 10.1182/blood-2018-99-117371
Abstract: Sickle cell disease (SCD) is an inherited blood disorder associated with a debilitating chronic illness. SCD is caused by a point mutation in the β-globin gene (HBB). A single nucleotide substitution converts glutamic acid to… read more here.
Keywords: scd hspcs; globin gene; target; gene edited ... See more keywords