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Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease

Archive ouverte | Ramadier, Sophie | CCSD

International audience. Sickle cell disease (SCD) is caused by a mutation in the b-globin gene leading to polymerization of the sickle hemoglobin (HbS) and deformation of red blood cells. Autologous transplantation ...

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

Archive ouverte | Weber, Leslie | CCSD

International audience. Sickle cell disease (SCD) is caused by a single amino acid change in the adult hemoglobin (Hb)  chain that causes Hb polymerization and red blood cell (RBC) sickling. The co-inheritance of m...

Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

Archive ouverte | Antoniou, Panagiotis | CCSD

International audience. Sickle cell disease and β-thalassemia affect the production of the adult β-hemoglobin chain. The clinical severity is lessened by mutations that cause fetal γ-globin expression in adult life ...

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