Article
Introducing a hemoglobin G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in mice.
Molecular therapy : the journal of the American Society of Gene Therapy - 4 Dec 2024
Li Chang, Georgakopoulou Aphrodite, Paschoudi Kiriaki, Anderson Anna K, Huang Lishan, Gil Sucheol, Giannaki Maria, Vlachaki Efthymia, Newby Gregory A, Liu David R, Yannaki Evangelia, Kiem Hans-Peter, Lieber André
Abstract excerpt
Precise repair of the pathogenic mutation in hematopoietic stem cells (HSCs) represents an ideal cure for patients with sickle cell disease (SCD). Here, we demonstrate correction of the SCD phenotype by converting the sickle mutation codon (GTG) into a benign G-Makassar variant (GCG) using in vivo base editing in HSCs. We show successful production of helper-dependent adenoviral vectors expressing an all-in-one...
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