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Gene Therapy for Genetic Diseases

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Thursday, May 20, 2021
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1:00 pm - 2:00 pm
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Dwight Koeberl, MD, PhD
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Duke CTSA Special Populations Spring Seminar Series

Dwight Koeberl, MD, PhD will describe the development of gene therapy for Pompe disease from proof of concept experiments to Phase I clinical trial initiation. This project was performed entirely in the academic environment at Duke University with federal resources, culminating in a partnership with an industry sponsor to support manufacturing and expansion of the clinical trial. It is one example of recent advances in the field of gene therapy for rare diseases.


Contact: Sydney Sullivan