Bahal, R., et. al. (2016) Nature Communications 7:13304.
Beta Thalassemia is a blood disorder caused by mutations in the HBB gene resulting in low levels of hemoglobin. As the genetic cause has been thoroughly characterized, it has been an attractive target for potential gene editing treatments, especially with the CRISPR/Cas9 explosion. However, clinically significant levels of gene editing have been difficult to achieve. Using a triplex-forming peptide nucleic acid coupled with a nanoparticle delivery system, Bahal et. al. were able to achieve a clinically relevant 7% gene editing in mice. This result demonstrates that gene editing techniques other than CRISPR/Cas9 may become clinically relevant.