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Principles of viral gene therapy for inherited pediatric disorders: Challenge and promise for copper transport disorders

by Stephen Gerard Kaler, MD, MPH, Professor of Pediatrics and Genetics, The Ohio State University College of Medicine


Professor Kaler is a renowned human geneticist and physician-scientist who was elected to the Association of American Physicians in 2014. He served as a postdoctoral fellow, clinical director, and tenured senior investigator in the National Institute of Health (NIH) intramural research program during a 25 year  career before retiring from the US Public Health Service and NIH.

In 2020, he joined the Center for Gene Therapy at Nationwide Children’s Hospital as a principal investigator and professor of pediatrics and genetics at The Ohio State University College of Medicine. Professor Kaler’s research, both in basic science and clinically, focuses on the study of inherited neurometabolic diseases in children. He emphasizes disorders of copper transport and lysosomal storage, specifically working on conditions like CTR1 deficiency, Menkes disease, and other ATP7A-related disorders, as well as alphamannosidosis. His laboratory is currently funded by NINDS/NIH to explore ATP7A viral gene therapy in combination with Copper Histidinate for Menkes disease.

As an active clinical investigator, Professor Kaler has led three natural history studies and four investigational new drug (IND) clinical trials. He has also been involved in nine other clinical protocols, including five that are currently ongoing. An influential mentor, he has trained 36 postdoctoral fellows or predoctoral students, with 15 of them receiving individual awards for research scholarship under his guidance. For his commitment to mentorship, he has been honoured with two Mentorship Awards from the NIH Intramural Research Programme.

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