The Gene Therapy Institute is 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. 


innovative industry-partnerships


faculty across 7 colleges


NIH funding awarded in FY2023

Gene Therapy Institute News

Image of brain scan during gene therapy injection
The future of gene therapy has arrived, and it's changing lives
A novel approach to gene therapy is improving lives in ways once thought impossible. Researchers at The Ohio State University Wexner Medical Center and College of Medicine have developed a new platform to deliver the gene therapy precisely to specific are
Image of man with skull in foreground
How gene therapy changed everything for child born with AADC deficiency
Ohio State researchers deliver life-changing treatment for genetic disorder
Scientist pipeting liquid into a test tube
Life-changing gene therapy expanding beyond neurological conditions
With treatments for conditions like Alzheimer’s disease on the horizon, researchers seek to address eye disease and chronic illness.
Dr. Krystof Bankiewicz, MD, PhD
$22 million in NIH award will accelerate 'CRISPR' research at UC Berkeley, Ohio State
The National Institutes of Health (NIH) is incentivizing researchers to think beyond single applications of genome editing and to develop platform approaches that can be used to address multiple diseases.
Photo of Krystof Baniewicz, Chief Scientific Officer of the Gene Therapy Institute
Gene therapy may offer new treatment strategy for alcohol use disorder
Gene therapy might offer a one-time, sustained treatment for patients with serious alcohol addiction, also called alcohol use disorder, according to a new study led by Krystof Bankiewicz, MD, PhD.
Kristy Townsend, PhD
Kristy Townsend, PhD, receives prestigious research award from W.M. Keck Foundation
The W.M. Keck Foundation awarded $1.2 million to a multi-center team, led by researchers at The Ohio State University College of Medicine and The Ohio State University Wexner Medical Center, to explore the neural feedback loop between the brain and adipos
Stephen Kolb, MD, PhD being interviewed for Ohio State Health Talks
Health Talks: The latest in ALS research at Ohio State
In this video, physician-scientist Stephen Kolb, MD, PhD, the latest discovery of a gene associated with ALS (amyotrophic lateral sclerosis) that will help explain how and why it causes the disease.
Photo of Russell Lonser, Director of the Gene Therapy Institute
De-Risking Cell And Gene Therapy Clinical Trials
Dr. Russell Lonser, Director of the Gene Therapy Institute, gives a historical overview and provides insight on planning for the future of gene therapy.
Illustration of gene therapy injection to the brain
New Gene Therapy Institute positions Ohio State as a leader in translational science
Ohio State holds one of the largest first-in-human clinical trial portfolios in gene therapy and is one of the only institutions with most of its gene therapy trials delivering targeted gene therapy to the midbrain.
Photo of Stephen Kolb, MD, PhD
Study examines the possible role of KIF5A gene in ALS
“Our findings could lead to a better understanding of how ALS develops in people who have this mutation,” said principal investigator Stephen Kolb, MD, PhD, professor of neurology and biological chemistry and pharmacology at Ohio State.
Krys Bankiewicz, Ohio state gene therapy researcher
Gene therapy is changing lives today; tomorrow, it could change millions
Ohio State scientist is studying a novel gene therapy model that could lead to treatment for widespread neurological diseases.
The oval at dawn
Ohio State launches new Gene Therapy Institute
The Institute will coordinate existing strengths to accelerate the expansion of therapies.

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