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Dr. Mueller is an expert researcher specializing in gene therapy and animal modeling of rare genetic disorders. He has had a distinguished academic research career, establishing himself as a thought leader in gene therapy and working on novel vector platforms, gene editing approaches, and mechanisms of gene silencing in the liver and CNS, authoring more than 75 publications and 10 patent applications.

He has worked on translating AAV-mediated gene therapies in alpha-1 antitrypsin deficiency, amyotrophic lateral sclerosis, Hermansky-Pudlak syndrome and Huntington’s disease. Notably, Dr. Mueller was the first to describe a regulatory T-cell response to the AAV capsid in patients, as well as the first to investigate AAV-mediated miRNA silencing in patients. Dr. Mueller was awarded tenure by the Department of Pediatrics at the University of Massachusetts Medical School in 2018. Currently, he serves as Head of Gene Therapy at Sanofi. A sought after speaker at national and international scientific meetings, Dr. Mueller has been the recipient of many honors and awards for his research, including the Bettina Irvine Family Award for Excellence in Alpha-1 Research, the Alpha-1 Foundation Shillelagh Award, the ALS Angel Fund Angel Award, and the Parker B. Francis Fellowship. In 2017, Dr. Mueller was honored for holding one of the top 10 patents in Massachusetts by the Boston Patent Law Association for inventing dual function AAV vectors with a silence and replace modality.

Dr. Mueller holds a B.A. from the Florida Institute of Technology, a Master’s in Clinical Investigation from the University of Massachusetts Medical School and a Ph.D. in Genetics from the University of Florida College of Medicine.