Fetal Gene Editing with Synthetic Nucleotides Cures Beta-Thalassemia in Mice

Scientists at Yale University and Carnegie Mellon University have used gene editing to effectively cure the genetic blood disorder β-thalassemia, in mouse fetuses, in utero.  The approach, which harnesses peptide nucleic acid (PNA) technology pioneered at Carnegie Mellon’s Center for Nucleic Acids Science and Technology (CNAST), corrected the disease-causing β-globin gene mutation, without any adverse effects on the fetus or pregnancy, and with no evidence of off-target effe