New Gene Editing Technology Cures Blood Disorder in Mice

Scientists from Carnegie Mellon University and Yale University have developed a next-generation gene editing system that has cured a genetic blood disorder in living mice using a simple intra-venous treatment.

The new system uses state-of-the-art peptide nucleic acid (PNA) molecules, a synthetic nucleotide technology that has been pioneered at Carnegie Mellon's Center for Nucleic Acids Science and Technology. It avoids previous issues encountered in other gene editing technologies such as CRISPR, which relies on DNA-cutting enzymes to slice open DNA at a target site to edit a specific gene. The new Carnegie Mellon/Yale system is designed to open up double-stranded DNA and bind near the target site in a highly specific manner without cutting cell's own DNA repair pathways to correct the malfunctioning hemoglobin subunit beta gene.

The new approach can help treat genetic diseases of the blood like beta thalassemia and sickle cell disease.

For more information, read the news release at the Carnegie Mellon University website.