Award supports researcher’s hunt to find a genetic cure for glaucoma

Gulab Zode, PhD, dreams of the day when glaucoma can be cured with a single shot. Support from the Research to Prevent Blindness foundation brings him a step closer to that day.

Zode has dedicated his career to studying the genetic causes of glaucoma. He specifically focuses on juvenile-onset glaucoma, a rare subset of the disease that affects children and teens and may lead to vision loss and blindness at an early age.

In both young and older patients, glaucoma’s mechanism is the same: fluid in the front of the eye does not flow freely through the trabecular meshwork and into the bloodstream. Instead, it builds up like water around a clogged drain, creating high pressure in the eye that can eventually damage the optic nerve and lead to vision loss.

“Often, these kids need multiple eye surgeries by age 30, and they still will eventually lose vision,” Zode explains. “If we can treat the root cause of the disease early on, they may never need additional glaucoma treatment. That’s not just saving money over their lifetime, it’s improving their quality of life.”

Previously, Zode discovered a genetic mutation in the myocilin gene that kills the healthy cells of the trabecular meshwork, essentially damaging the eye’s drainage system and increasing eye pressure. Because it causes 30% to 50% of juvenile glaucoma cases, this mutation is an ideal target for emerging gene-editing treatments.

Zode has proposed using the Nobel Prize-winning gene-editing technology CRISPR/CAS9 to remove the genes that produce the mutated myocilin in the eye. To deliver that gene editor to the cells in the eye, he proposes using lipid nanoparticles provided by Philip Felgner, PhD, a UC Irvine School of Medicine professor of physiology and biophysics. Feigner’s pioneering work in gene editing helped contribute to the development of mRNA vaccines, including the vaccines developed to combat COVID-19.