While CRISPR-Cas9 has brought genome editing to the masses, it’s not the only genome editor in town. Dr. Peter Glazer will discuss his work to develop peptide nucleic acids (PNAs) into a robust tool for targeted DNA engineering. PNAs bind to DNA double helices to form a triple helix, recruiting DNA repair proteins and facilitating incorporation of new DNA segments. By delivering PNAs and donor DNA to hematopoietic stem/progenitor cells (HSPCs) in biodegradable nanoparticles, Glazer and his team are able to correct mutations in the beta-globin gene. This intriguing approach is an attractive alternative to genome editing methods that involve DNA cleavage.
Genome Editing in Adult and Fetal Mice by PNA Nanoparticles
Professor, Therapeutic Radiology & Genetics
Yale University School of Medicine