Developing a gene therapy to fix broken bones

What biomedical issue did you address in your research, and what did your studies find? 

My Ph.D. research focused on developing a gene therapy approach to stimulate bone regeneration in "nonunion fractures," which occur when broken bones fail to heal naturally. I designed and tested viral vectors that deliver therapeutic genes directly to the fracture site, aiming to restart the biological processes that drive bone repair. This therapeutic strategy helps create an environment where bone-forming cells can resume the repair process when normal healing has stopped. 

In addition, I investigated how a combination of immune-stimulating growth factors can improve the quality of regenerated bone so that it more closely resembles the body's natural bone. By combining laboratory experiments, preclinical testing, and close collaboration with clinicians, I demonstrated the potential of this gene therapy approach as a future treatment for patients with nonunion fractures.

What aspects of your training at Mayo helped you grow as a scientist and as a thinker?  

I engaged in frequent discussions with clinicians, which helped ground my scientific ideas in real clinical needs and showed me how closely research can connect to patient care. These interactions pushed me to think beyond whether a therapy could work biologically and instead consider how it would realistically function within a clinical setting. 

The training environment at Mayo Clinic Graduate School of Biomedical Sciences also fostered a strong foundation in both laboratory and translational research. The graduate school's support for my co-mentored thesis across two laboratories and departments strengthened my ability to collaborate effectively, communicate across disciplines and appreciate the importance of teamwork in solving complex scientific problems.

What's next?

I am continuing my work as a postdoctoral fellow in the Department of Orthopedics at Mayo Clinic to further advance the gene therapy platform I developed during my thesis and explore its potential for nonunion fractures and additional regenerative applications.

This article was written by Meredith Lilley, a Ph.D. candidate in Neuroscience at Mayo Clinic Graduate School of Biomedical Sciences.