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Dr. Gonzalez-Alegre's long-range research goal is to advance the application of precision medicine in the neurology clinic. His research focus revolves around genetic disorders that affect the brain, spanning from the diagnosis of novel genetic disease in the clinic to the identification of novel molecular targets using disease models and the design of early-phase human clinical trials.
Dr. Gonzalez-Alegre is a clinician-scientist devoted to the investigation and clinical care of inherited neurological disorders, particularly those that affect movement. His laboratory focuses on gaining a better understanding of the neurobiological bases of the movement disorder dystonia, aiming to develop novel therapeutic approaches.
Dr. Gonzalez-Alegre’s lab has made important contributions to the understanding of the most common form on early onset inherited dystonia, known as DYT1. Those include addressing the role of the mutated protein, torsinA, in the endoplasmic reticulum and nuclear envelope, using cellular and molecular approaches in cell-based and animal models of the disease. He has also pioneered the development of gene silencing strategies as a potential therapeutic approach for DYT1. More recently, the focus of Dr. Gonzalez-Alegre’s research has expanded to other genetic forms of dystonia, such as DYT6 or X-linked dystonia parkinsonism, among others. The goal of these new studies is to identify common pathogenic mechanisms shared by different genetic forms to converge on a single therapeutic approach for all.
As director for Clinical Programs at the Raymond G. Perelman Center for Cellular and Molecular Therapy at Children’s Hospital of Philadelphia, Dr. Gonzalez-Alegre participates in the design of preclinical, investigational new drug-enabling and early phase human trials for inherited brain diseases, interacting with many other members of the team that span from basic scientist to experts of regulatory affairs. In addition, he directs a Huntington’s Disease (HD) Center of Excellence at the University of Pennsylvania, where he conducts research in HD patients involving neuroimaging, the use of biosensors for objective phenotyping or clinical trials, among others.
Education and Training
MD, University of Malaga Medical School, 1996
PhD, University of Malaga Medical School (Neuroscience), 2009
Titles and Academic Titles
Director for Clinical Programs, Raymond G. Perelman Center for Cellular and Molecular Therapeutics
Associate Professor of Neurology
American Academy of Neurology, 1999-
Movement Disorders Society, 2000-
American Neurological Association, 2010-
S. Weir Mitchell Award, American Academy of Neurology, 2003
Junior Award for Excellence in Basic Science Research, Movement Disorders Society, 2004
Medical Education Faculty Award, University of Iowa College of Medicine, 2009, 2012
Stanley Fahn Award, Dystonia Medical Research Foundation, 2012
Jon Stolk Award in Movement Disorders for Young Investigators, American Academy of Neurology, 2012
Resident Educator of the Year Award, Department of Neurology, the University of Iowa, 2014