Gene Therapy

Gene therapy is a process of modifying the genetic code of diseased cells to improve disease outcomes. OSU has established a team to provide expert services in diagnosis, genetic counseling, and gene therapy clinical trials. The OSU Inherited Retinal Disease (IRD) team also studies innovative ways to treat a wider variety of IRDs with effective gene therapy.

Thomas Mendel, MD, PhD

Genetics research is essential for precision-based care to provide earlier diagnosis, improve outcomes, elucidate the genetic causes of disease, and determine the best treatments and their timing for each patient. Thomas Mendel, MD, PhD, has built a genetics program to develop and implement gene therapies in patients with inherited retinal eye diseases. Common symptoms include diminished vision in low light or night blindness, loss of color vision, light sensitivity, and vision loss.

Patients with IRDs slowly lose vision due to damaged cells in the retina. Dr. Mendel provides care to both adults and children, who can be affected by these diseases, in the Department of Ophthalmology & Visual Sciences at the Ohio State and Nationwide Children's Hospital.

A clinician-scientist, Dr. Mendel joined Ohio State in August 2021. He's leveraging his 15 years of training and expertise to build both an IRD focused clinical team that can provide the very latest diagnosis and evaluation for emerging IRD clinical trials.  He also has established a translational gene therapy lab to accelerate new gene-based treatments for inherited retinal diseases.

“This is the first time in human history that we can address these diseases,” Dr. Mendel says. “In the past, we could diagnose these conditions, but we could not improve them.”

Genetic Counseling

Using Genetics to Reverse and Prevent Neurodegenerative Vision Conditions

Genetics research is essential for precision-based care to provide earlier diagnosis, improve outcomes, explain the genetic causes of disease and determine the best treatments and their timing for each patient.

Patients with IRDs can experience diminished vision in low light or night blindness, loss of color vision, light sensitivity and progressive vision loss due to degeneration of specific retinal cells.

Dr. Mendel partners with other researchers at Ohio State and at Nationwide Children’s Hospital, which houses the Center for Gene Therapy. There they run a dynamic laboratory that explores their patent-pending technology to expand gene therapy to more IRDs and deliver it with less trauma and inflammation to the retina. 

“We have built from scratch a large, full surgery suite for preclinical testing and an experimental platform for retinal surgery and testing gene therapy more robustly for multiple conditions,” Dr. Mendel says. 

Dr. Mendel’s education includes lab training and work in stem cell therapy for diabetic retinopathy at the University of Virginia, followed by ophthalmology residency at Vanderbilt University, then a vitreoretinal surgery fellowship at Cleveland Clinic’s Cole Eye Institute, and pediatric vitreoretinal post-fellowship training at University of Michigan's Kellogg Eye Center.

Gene Therapy Institute

Dr. Mendel’s laboratory is housed within OSU’S Gene Therapy Institute, an interdisciplinary community bringing together world-class faculty with diverse expertise in the area of gene therapy research. Through collaboration we are enabling the successful translation of basic science discoveries into new therapies that are accessible to affected populations around the globe. 

The rapid expansion and use of gene therapy globally have made it critical to organize, structure and strengthen efforts in this fast-moving field. The Gene Therapy Institute was formed to support these critical administrative, recruiting and infrastructural needs. Our faculty have uniquely positioned the Institute as a global leader in this area, with end-to-end capabilities ranging from fundamental science to clinical trials and, ultimately, commercialization.

Gene Therapy Institute

The Big Picture: Marrying Research and Patient Care

Dr. Mendel’s focus on IRD both in translational research and clinical care helps to serve Ohio State as it continues to its ophthalmology research. Dr. Mendel says it’s incredibly powerful to see children respond to the therapy. After surgery, many go from being nearly blind with no night vision to having vision between 20/40 and 20/70.

“You see them perk up and respond to visual cues for the first time, asking me questions about the details of their own retina photos on the computer screen. It’s pretty moving,” Dr. Mendel says.

As a clinician, Dr. Mendel is motivated by his patients. Dr. Mendel says his lab will greatly benefit from the recently awarded National Institutes of Health P30 Center Core Grant, called The Ohio State University Vision Sciences Research Core Program. “The P30 core grant reflects on a national scale some of the pieces that Sayoko Moroi, MD, PhD, chair of the Department of Ophthalmology and Visual Sciences, has already put in place, richly expanding research expertise and investment in quality to accelerate our work,” Dr. Mendel says.

“I benefit directly from many facets of the P30,” he says. “One of the main reasons I came to Ohio State was because the job with these resources was too good to pass up. Dr. Moroi has pulled together all the things that make for successful research — mentorship, camaraderie, and teamwork.

“We are at the very beginning of a new wave of medicine in ophthalmology and retina care."

The Pelotonia Research Center is the first new building to open at Carmenton, Ohio State's Innovation District. This five-story, $228 million, 305,000-square-foot laboratory building is equipped with new spaces, technologies and resources needed for researchers to work across disciplines to accelerate new discoveries. Both the OSU Gene Therapy Institute and much of the Department of Ophthalmology’s basic research are housed on the expansive 4th floor of the Pelotonia Research Center.

This allows Dr. Mendel and others to be together and collaborate across various ophthalmology projects. Current and future faculty recruits will further develop and enhance the Precision Based Vision Care and Research Programs of Excellence with an emphasis in diabetic retinopathy, glaucoma, retinal detachments, corneal dystrophies, eye cancers, eye genetics, vision impairment - low vision and mobility, and population health. This unique environment will house the latest imaging equipment and will allow our established and emerging faculty to make new discoveries. We will translate these discoveries to develop innovative new treatments for both common and complex eye conditions and improve healthcare for all. We are eager to create a better future for our patients and embrace our mission: Restore, Preserve, and Enhance Vision to Improve Lives for All.