Dr. Dwight Koeberl

Biography

Dwight D. Koeberl is a physician-scientist specializing in clinical biochemical genetics and gene therapy. His research focuses on developing therapies for inherited metabolic disorders, particularly through the use of adeno-associated virus (AAV) vectors and genome editing techniques such as CRISPR/Cas9. He has pioneered gene therapy for glycogen storage diseases (GSD) and phenylketonuria (PKU). Koeberl's work includes developing AAV8-pseudotyped vectors to treat GSD Ia by correcting glucose-6-phosphatase deficiency in animal models. He also investigates gene therapy approaches to address enzyme replacement therapy challenges in Pompe disease, including mitigating antibody responses. Koeberl has successfully collaborated on gene therapy experiments that have shown long-term biochemical corrections for PKU in mouse models. His ultimate goal is to develop effective genome editing strategies that can treat glycogen storage diseases at an early stage, thereby offering long-term health benefits.

Research Interests

Experience