Pedro Gonzalez-Alegre, MD, PhD

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.