FAU researchers receive $1.9 million NEI grant for degenerative disease treatment research

Lisa A. Brennan, PhD, research professor (left), and Marc Kantorow, PhD, associate dean of graduate programs and a professor of biomedical science, both of FAU Schmidt College of Medicine. Image credit: Florida Atlantic University Schmidt College of Medicine.

Florida Atlantic University’s (FAU) Schmidt College of Medicine researchers received a 5-year, $1.9 million grant from the National Eye Institute (NEI) for the treatment of degenerative disease by replacement of dysfunctional cells with new cells.¹ The research team, led by principal investigator Marc Kantorow, PhD, associate dean of graduate programs and a professor of biomedical science, and Lisa A. Brennan PhD, research professor at the FAU Schmidt College of Medicine, will work to determine the novel mechanisms that convert immature eye lens precursors cells into functional transparent cells, according to a news release.

These replacement cells can be genetically engineered in FAU’s laboratory through programming of embryonic stem cells that can act as universal precursors for formation of mature cells and tissues. However, a proven point of difficulty has been identifying all the requirements for adult cell formation.¹

“This research led by the Kantorow laboratory is a game-changer for the future of regenerative medicine,” said Lewis S. Nelson, MD, dean and chief of health affairs at the FAU Schmidt College of Medicine, in the release. “By uncovering the precise requirements for transforming stem cells into fully functional adult cells, we are addressing one of the most critical challenges in treating degenerative diseases. This breakthrough has the potential to dramatically improve patient outcomes by offering new hope for restoring damaged tissues and organs, ultimately enhancing both the quality and longevity of life.”

Kantorow’s laboratory team had previously discovered that low oxygen levels (hypoxia) are a key driver of the cellular reprogramming events leading to formation of mature transparent lens cells.¹ The new grant will allow the team to identify pathways controlling this hypoxia-induced cellular transformation event, the release stated.

“Specifically, our work will examine the role played by the master regulator of the hypoxic response, hypoxia-inducible transcription factor 1 or HIF1a in control of lens gene expression and the requirement for oxygen-induced epigenetic modifications that regulate DNA conformation,” said Kantorow in the release. “These novel studies, using lens cells as a model system, provide clues into the development of strategies for engineering of more complex cells and tissues and therefore novel therapies for the treatment of degenerative disease.”

“While signaling molecules like fibroblast growth factors are known to start this transformation, we still don’t fully understand how critical genes for fiber cell maturation are regulated,” said Brennan in the release. “Our latest research aims to explore how hypoxic conditions in the lens core help drive gene activation through unique transcriptional and epigenetic mechanisms, filling a key gap in our knowledge about lens development.”

The Kantorow laboratory will focus on understanding the molecular and mitochondrial mechanisms that power ocular development and disease. The lab uses the eye lens and retina as model system to explore cellular differentiation, function, and disease processes, with research spanning from analyzing the roles of genes implicated in ocular diseases to study the direct effects of environmental factors on mitochondrial and cellular functions. Researchers also explores how key cellular systems can be manipulated to prevent or treat ocular conditions, including age-related cataract formation and macular degenerations.¹

“Hypoxia activates key transcription factors and induces chromatin changes, regulating a wide array of cellular processes, including autophagy and cellular remodeling,” said Kantorow in the release. “Findings from our studies not only illuminate fundamental biological mechanisms of cellular differentiation but also provide a foundation for developing regenerative therapies and other treatments for age-related and degenerative diseases.”

Reference:

1. Galoustian G. FAU lands $1.9 million NIH grant for novel way to treat degenerative disease. Florida Atlantic University. February 4, 2025. Accessed March 13, 2025. https://www.fau.edu/newsdesk/articles/nih-grant-degenerative-disease#:~:text=Researchers%20from%20Florida%20Atlantic%20University,dysfunctional%20cells%20with%20new%20cells.