Nanomedicine Center for Nucleoprotein Machines

The Nanomedicine Center for Nucleoprotein Machines focuses on understanding and re-directing natural processes for repair of damaged DNA. Human cells have many different repair pathways, each of which involves a different type of nucleoprotein machine. The Center’s five-year goal is to re-engineer the homologous recombination repair machine to provide a clinically applicable gene correction technology. The Center’s vision is to design, produce, deliver, and validate a gene correction device based on engineered zinc finger nucleases. The device will home to the defective gene in the patient’s hematopoietic stem cells and make a precise cut to activate the HR machine, which will replace the mutation with the correct beta-globin sequence. The approach can potentially be extended for treatment of other single-gene disorders.