Article
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
The Journal of clinical investigation - 3 Oct 2016
Brendel Christian, Guda Swaroopa, Renella Raffaele, Bauer Daniel E, Canver Matthew C, Kim Young-Jo, Heeney Matthew M, Klatt Denise, Fogel Jonathan, Milsom Michael D, Orkin Stuart H, Gregory Richard I, Williams David A
Abstract excerpt
Reducing expression of the fetal hemoglobin (HbF) repressor BCL11A leads to a simultaneous increase in γ-globin expression and reduction in β-globin expression. Thus, there is interest in targeting BCL11A as a treatment for β-hemoglobinopathies, including sickle cell disease (SCD) and β-thalassemia. Here, we found that using optimized shRNAs embedded within an miRNA (shRNAmiR) architecture to achieve ubiquitous...
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