Dwight D. Koeberl, MD, PhD

Dwight D. Koeberl, MD, PhD
Co-Director, Integrating Special Populations Core

Contact Information:

Professor of Pediatrics

The focus of our research has been the development of gene therapy with  adeno-associated virus (AAV) vectors, most recently by genome editing with CRISPR/Cas9. We have developed gene therapy for inherited disorders of metabolism, especially glycogen storage disease (GSD) and phenylketonuria (PKU).