Gene Therapy Program Update May 20, 2026

Over the past three years, MAODF has been working with research teams at University of Texas Southwestern Medical Center and The Ohio State University to develop a gene therapy approach for patients with Monoamine Oxidase Deficiency (MAOD). Gene therapy is considered a promising long- term strategy because it may one day provide a more permanent correction of the underlying disease.

In the early stages of the project, researchers tested a commonly used AAV9 gene therapy delivery system together with an MAOD mouse model developed from the laboratory of Jean Shih in California. Although these studies provided important information, the mouse model did not consistently show the behavioral features needed to fully evaluate treatment success. To improve the research, Jackson Laboratory recently developed a new MAOD mouse model that appears to better reflect the disease. However, the new model is proving difficult to breed in large numbers required for treatment studies.

At the same time, advances in the gene therapy field continue to provide new hope. Researchers at the Broad Institute (MIT and Harvard) have recently developed newer AAV capsids that may improve delivery of gene therapies to the brain and central nervous system. MAODF is therefore exploring the possibility of restarting its gene therapy program in the future using these next-generation delivery systems together with improved MAOD animal models developed through ongoing research collaborations.