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Contact
E-mail
gonzalezap [at] email.chop.edu
Phone
Location - People View
Room 5062

3501 Civic Center Blvd
Philadelphia, PA 19104
United States

Research Topics
Pedro Gonzalez-Alegre, MD, PhD
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.
Director for Clinical Programs, Raymond G. Perelman Center for Cellular and Molecular Therapeutics

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.

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Bio

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

Professional Memberships

American Academy of Neurology, 1999-

Movement Disorders Society, 2000-

American Neurological Association, 2010-

Professional Awards

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

Publication Highlights

Beauvais G, Rodriguez-Losada N, Ying L, Zakirova Z, Watson JL, Readhead B, Gadue P, French DL, Ehrlich ME, Gonzalez-Alegre P. Exploring the Interaction Between eIF2α Dysregulation, Acute Endoplasmic Reticulum Stress and DYT1 Dystonia in the Mammalian Brain. Neuroscience. 2018 Feb; 371:455-468. PMID: 29289717
Zakirova Z, Fanutza T, Bonet J, Readhead B, Zhang W, Yi Z, Beauvais G, Zwaka TP, Ozelius LJ, Blitzer RD, Gonzalez-Alegre P and Ehrlich ME. Mutations in THAP1/DYT6 reveal that diverse dystonia genes disrupt similar neuronal pathways and functions. PLoS Genetics. 2018 Jan; 14(1):e1007169. PMID: 29364887
Beauvais G, Bode N, Watson J, Wen H, Kawano H, Glenn KA, Harata NC, Ehrlich ME, and Gonzalez-Alegre P. Disruption of protein processing in the endoplasmic reticulum of DYT1 knock in mice implicates novel pathways in dystonia pathogenesis. J Neurosci. 2016 Oct; 36(40):10245-10256. PMID: 27707963
Gonzalez-Alegre P, Bode N, Davidson BL, Paulson HP. Silencing primary dystonia: lentiviral-mediated RNA interference therapy for DYT1 dystonia. J Neurosci. 2005 Nov; 25: 10502-10509. PMID: 16280588
Gonzalez-Alegre P, Paulson HP. Aberrant cellular behavior of mutant TorsinA implicates nuclear envelope dysfunction in DYT1 dystonia. J Neurosci. 2004 Mar; 24(11):2593-2601. PMID: 15028751