Nanomedicine is the field concerned with characterization and manipulation of biological processes on the nanometer scale.

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 will test the efficacy of its approach in a mouse model of Sickle Cell Disease, which is caused by a single (A to T) mutation in the beta-globin gene. The disease is painful and life shortening, and there is no general cure. In the US, Sickle Cell Disease afflicts primarily persons of African origin and thus contributes to minority health disparities.

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.

We are one of six nanomedicine development centers supported by the US National Institutes of Health Common Fund