Single injection treats hemophilia B for life, in proof-of-concept study -- MEDICA - World Forum for Medicine

Single injection treats hemophilia B for life, in proof-of-concept study

02/05/2018

Image: Hepatocyte-like cells (HLCs) differentiated from the stem cells of patients with hemophilia B show very low levels of clotting factor IX, or FIX, shown in white; Copyright: Salk Institute

Left two panels: Hepatocyte-like cells (HLCs) differentiated from the stem cells of patients with hemophilia B show very low levels of clotting factor IX, or FIX, shown in white. Right two panels: After treating these same cell lines with a gene correction tool, FIX increased to healthy levels.