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Kathrin C. Meyer, PhD

Principal Investigator
Nationwide Children’s Hospital
700 Childrens Drive
Columbus, Ohio, United States

Kathrin C. Meyer, PhD, studied Cellular and Biomedical Sciences at the Institute of Cell Biology in Berne, Switzerland. Her Post-Doctoral research was performed in Brian Kaspar’s laboratory at the Center for Gene Therapy in Columbus Ohio. During that time, Dr. Meyer established a new and fast reprogramming method for in vitro modeling of neurodegenerative diseases using patient skin cells. Moreover, Dr. Meyer developed intrathecal gene therapy programs for several neurodegenerative diseases including Spinal Muscular Atrophy and Batten Disease. Dr. Meyer became a Principal Investigator at Nationwide Children’s Hospital. She is also an Assistant Professor in the Department of Pediatrics at The Ohio State University, Columbus, Ohio.

Dr. Meyer’s lab focuses on studying disease mechanisms in neurodegenerative diseases and finding new innovative therapeutic strategies for them. Her main emphasis is understanding the impact of different mutations on disease course and severity. Moreover, Dr. Meyer’s lab has a strong translational focus in her projects with the goal to move additional programs towards clinical trials. These projects include the optimization of delivery strategies and evaluation of efficacy and targeting of various areas of the nervous system with gene therapy.


Representative Publications:

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Improving Single Injection CSF Delivery of AAV9-Mediated Gene Therapy for SMA: A Dose-Response Study in Mice and Nonhuman Primates

Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept

Onasemnogene Abeparvovec-Xioi: A Gene Replacement Strategy for the Treatment of Infants Diagnosed With Spinal Muscular Atrophy


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