Browsing: nanoparticles

In the not-yet-peer-reviewed study, the researchers targeted the erythroid-specific +58 kb enhancer of BCL11A, whose disruption relieves repression of γ-globin and raises fetal haemoglobin. They treated primary CD34+ HSPCs from three healthy HbAA donors and two donors with sickle cell disease using either Cas9-sgRNA ribonucleoprotein electroporation or lipid nanoparticles carrying Cas9 mRNA and the same…