Buckeye engineers receive $1.2M grant to advance neurofibroma therapies
Biomedical Engineering Assistant Professor Natalia Higuita-Castro received a three-year, $1.2 million grant from the Gilbert Family Foundation to develop next-generation gene therapies to treat neurofibromas.
Affecting approximately 2 million people worldwide, neurofibromatosis is a genetic disorder that causes tumors—called neurofibromas—to grow along the nerves in the body or under the skin. Usually diagnosed in childhood, NF1 is the most common of the three types of neurofibromatosis.
While generally not cancerous, neurofibromas can cause significant health issues such as blindness, cancer, hearing loss, bone deformities and cardiovascular issues. More than half of all NF1 patients also suffer from learning disabilities. The course of the disease is unpredictable and varies in severity among individuals.
Higuita-Castro and co-investigator Daniel Gallego-Perez, an associate professor of biomedical engineering and surgery, will apply novel nanotechnology and gene-delivery approaches to treat NF1. Specifically, they will develop custom-made extracellular vesicles (EVs) capable of targeting neurofibromas to deliver genetic material to reduce the number of tumors in the body.
According to Higuita-Castro, since EVs are natural, cell-derived carriers that play a crucial role in cell-to-cell communication, their use in delivering therapeutics minimizes inflammatory immune system response versus other biological delivery systems, like viruses.
“Using designer EVs as novel delivery systems for the treatment of NF1 will help overcome major limitations observed with other delivery methodologies,” she explained. “Currently there are no examples of EV-based carriers for the deployment of gene therapies against NF1. Thus, the work proposed herein is fundamentally innovative and potentially transformative.”
If successful, the advancements derived from this research will allow the use of new, personalized treatments against NF1, Higuita-Castro said. These advances will be key in enabling future research collaborations to help find an effective treatment for the condition.
The team will also utilize tissue nanotransfection, a process developed at The Ohio State University by Gallego-Perez, to generate the custom extracellular vesicles loaded with the therapeutic genes in situ. This technology can be used to engineer cells for treatment within the patient’s own body.
Higuita-Castro and Gallego-Perez have conducted various individual research projects on the application of custom extracellular vesicles and tissue nanotransfection as therapeutics to treat a range of conditions and diseases, including brain tissue damaged by a stroke, low back pain, diabetes and Alzheimer’s disease.
This is the first Gilbert Family Foundation grant to support Ohio State research and is part of more than $18 million in grants awarded recently as part of the organization’s Gene Therapy Initiative. The private, non-profit foundation aims to accelerate the development of curative therapies that address the underlying genetic abnormalities in neurofibromatosis type 1 (NF1) patients.
by Candi Clevenger, College of Engineering Communications, clevenger.87@osu.edu
