WVU undergraduate student explores gene therapy approaches to treating blinding diseases through summer research program

Summer break can mean a lot of different things for students – taking part-time jobs, returning to their hometowns and relaxing under the sun after a challenging academic year. But for Kenneth Wang, a senior biomedical engineering and biochemistry major at West Virginia University, summer meant taking steps toward treating the blinding disease known as blue cone monochromacy.

Wang participated in the Summer Undergraduate Vision Research Fellowship Program, a 10-week, full-time vision research-focused internship supported by faculty in the Departments of Ophthalmology and Visual Sciences and Biochemistry and Molecular Medicine.

The highly selective program provides undergraduate students with basic biochemistry and visual neuroscience research experience in areas that explore blinding diseases and new diagnostic treatment methods.

Having completed the University’s Summer Undergraduate Research Experience (SURE) in 2023, Wang said he was eager to explore another program where he could spend the summer working alongside WVU researchers on major research projects.

“By pairing us with faculty members who are constantly engaged in research, we as students can build on our skillset while making valuable contributions to groundbreaking studies aimed at better understanding and treating blinding diseases,” he said.

Wang was partnered with Wen Tao Deng, Ph.D., assistant professor for the Departments of Biochemistry and Molecular Medicine and Ophthalmology and Visual Sciences, and Brooke Brothers, a doctoral student in Dr. Deng’s lab, on a project that explores how a gene therapy approach could be implemented to treat the retinal disease known as blue cone monochromacy.

“BCM is a rare, genetic disorder that causes severe color blindness, day blindness, poor visual acuity, involuntary eye movement and photophobia,” Wang explained. “Our research aims to determine if a gene therapy approach could be utilized to treat certain BCM mutations, specifically one that expresses a mutant biomolecule.”

Wang explained that previous studies have found that it is possible to treat other BCM mutations in laboratory models that don’t express these mutant biomolecules, so he and his team set out to determine if this method would be viable when these molecules are present.

To explore this hypothesis, Wang said he spent the majority of the program’s 10-week duration focusing on cell culture —the growth of cells in a controlled laboratory setting outside their natural environment —and on a unique process known as plasmid modification.

“Plasmids are genetic structures in cells that are capable of replicating independently from a cell’s main chromosome,” he explained. “They act as ‘vehicles’ for carrying and copying specific pieces of DNA, making them extremely useful for genetic engineering. In the lab, we can modify these plasmids by altering their DNA, ultimately changing their function, and observe those results in real time.”

Although the program concluded in August, Wang said he is still continuing this research alongside well into the fall semester, and the findings so far present a promising future for patients who are suffering from BCM.

“Our work is still ongoing and there is much left to do, but we have progressed from our starting plasmid to one we can now test in human cells in the lab. We hope that this research will be foundational in receiving approval for human trials, bringing us closer to our goal of helping patients in need preserve and protect their vision.”

Wang explained that the next steps for this research are to continue testing the effectiveness of these plasmids in cell cultures and eventually in laboratory models that mimic the human BCM condition.

“I am grateful to this program for allowing me to play a role in such important research,” he said. “This program grants students like me the rare opportunity to spend extended time in a laboratory setting, planning and executing experiments, and analyzing complex data. It helps give us a taste of what a career in research can be like, while providing an ideal environment for us to grow and build on our research skills.”

To learn more about vision research at WVU, visit medicine.wvu.edu/ophthalmology-and-visual-sciences/research.