Miami Project Receives NIH Grant to Study Axon Regeneration

University of Miami Miller School of Medicine Principal Investigators Kevin Park, Ph.D. Associate Professor, Department of Neurological Surgery and The Miami Project, Vance Lemmon, Ph.D., the Walter G. Ross Distinguished Chair in Developmental Neuroscience and Professor, Department of Neurological Surgery and The Miami Project, Sanjoy Bhattacharya, Ph.D., Professor of Ophthalmology, were awarded a $2 million grant from the National Institutes of Health (NIH) over three years to study Novel targets to promote RGC axon regeneration: Insights from unique retinal ganglion cell cohorts.

NIH will fund a total of six projects to identify biological factors that affect neural regeneration in the retina and the Miller School team was the recipient of one. The projects are part of the National Eye Institute (NEI) Audacious Goals Initiative (AGI), a targeted effort to restore vision by regenerating neurons and their connections in the eye and visual system.

Drs. Park and Lemmon are using RNA sequencing in mouse retinal ganglion cells to identify differences in the expression of genes in regenerative versus non-regenerative retinal ganglion cells. In parallel, Park and Bhattacharya will use mass spectrometry to determine what lipids (or fat molecules) may give subclasses of retinal ganglion cells more robust regenerative capacities. The researchers will then perform a set of experiments aimed at understanding the function of the genes found to be involved in regeneration. The most promising gene candidates will be used as a therapy aimed at regenerating the optic nerve in a mouse model with optic nerve injury.

“Some central nervous system neurons can regenerate their axons while others cannot. We know very little about the mechanisms that give rise to such a difference in axon regeneration capacity. If one can identify what allows regeneration in these unique neurons, we could potentially promote strong regeneration in neurons in general,” said Dr. Park.

Most irreversible blindness results from the loss of neurons in the retina, which is the light-sensitive tissue in the back of the eye. Many of the common eye diseases including age-related macular degeneration, glaucoma and diabetic retinopathy, put these cells at risk. Once these neurons are gone, humans have little if any capacity to replace them. These are central nervous system cells and, if successful, could provide applications for other neurodegenerative diseases, disorders and injuries like spinal cord and brain injuries.

“Understanding factors that mediate the regeneration of neurons and the growth of axons is crucial for the development of breakthrough therapies for blinding diseases. What we learn through these projects will have a health impact beyond vision,” said Paul A. Sieving, M.D., Ph.D., director of NEI, part of NIH.

This project is one of six funded by NIH that hopes to add to the knowledge base from several recent key advances. Researchers recently reported a technique that increases the regenerative capacity of retinal axons in a mouse model of optic nerve injury, a model commonly used to study glaucoma and other optic neuropathies. Progress also has been made in identifying factors that either stimulate or inhibit regeneration of neurons required for vision. The newly-funded projects will further this area of axon regeneration research by identifying cues that guide axons to appropriate targets in the brain, allowing functional connections to re-establish between the eye and the visual processing system.

For more information about these and other AGI projects, visit NEI AGI.

NEI leads the federal government’s research on the visual system and eye diseases. NEI supports basic and clinical science programs to develop sight-saving treatments and address special needs of people with vision loss.

About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases.