Listar por tema "Anemia, Sickle Cell"
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Evidence for the molecular heterogeneity of sickle cell anemia chromosomes bearing the βS/benin haplotype
(2005)There are at least four distinct African and one Asian chromosomal backgrounds (haplotypes) on which the sickle cell mutation has arisen. Additionally, previous data suggest that the βS/Bantu haplotype is heterogeneous at ... -
In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies
(2022)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 ...