[PDF][PDF] Base editing of gamma globin gene promoters generates durable expression of fetal hemoglobin for the treatment of sickle cell disease
AP Rybak, EZ Akrawi, C Rinaldi, SJ Haskett, L Lin… - Mol. Ther, 2020 - beamtx.com
… with Gamma Globin Protein Induction In Vitro • Linear regression analysis demonstrates
that HBG1/2 gene promoter base editing and gamma globin protein levels in human
erythroid cells in vitro are tightly correlated. … • Similar human chimerism, HBG1/2 promoter
base editing and gamma globin protein upregulation has been achieved in a second
mobilized CD34+ HSPC donor at 18 weeks post-transplantation. …
that HBG1/2 gene promoter base editing and gamma globin protein levels in human
erythroid cells in vitro are tightly correlated. … • Similar human chimerism, HBG1/2 promoter
base editing and gamma globin protein upregulation has been achieved in a second
mobilized CD34+ HSPC donor at 18 weeks post-transplantation. …
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