In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies
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DOI: 10.1172/jci.insight.162939
Abstract: Individuals with β-thalassemia or sickle cell disease and hereditary persistence of fetal hemoglobin (HPFH) possessing 30% fetal hemoglobin (HbF) appear to be symptom free. Here, we used a nonintegrating HDAd5/35++ vector expressing a highly efficient… read more here.
Keywords: editing single; vector; base editing; vivo base ... See more keywords