Description of Research Expertise:
Dr. Gonzalez-Alegre is a highly committed clinician-scientist who directs a laboratory devoted to the investigation of the neurobiological bases and therapeutics of dystonia. For his research efforts, funded through federal and private sources, he has been recognized with 2003 S Weir Mitchell Award of the American Academy of Neurology, the 2004 Junior Award for Excellence in Basic Science Research from the Movement Disorders Society, the 2012 Jon Stolk Award in Movement Disorders from the American Academy of Neurology, and the 2012 Stanley Fahn Award from the Dystonia Medical Research Foundation. He is also Co-Director for Clinical Programs of the Raymond G. Perelman Center for Cellular & Molecular Therapy at the Children Hospital of Philadelphia, involved in translational efforts to advance novel molecular therapies for various neurological diseases to early phase clinical trials.
Selected Publications:
Gonzalez-Alegre P.: Advances in cell biology of dystonia: Focus on torsinA Neurobiology of Disease 127 : 233-241,2019.
Narendra DP, Isonaka R, Nguyen D, Schindler AB, Kokkinis AD, Ehrlich D, Bardakjian TM, Goldstein DS, Liang TW, Gonzalez-Alegre P.: Peripheral synucleinopathy in a DJ1 patient with Parkinson disease, cataracts, and hearing loss. . Neurology 92 (23): 2019.
Beauvais G, Watson JL, Aguirre JA, Tecedor L, Ehrlich ME, Gonzalez-Alegre P.: Efficient RNA interference-based knockdown of mutant torsinA reveals reversibility of PERK-eIF2α pathway dysregulation in DYT1 transgenic rats in vivo. Brain Research 1706 : 24-31,2019.
Bardakjian TM, Faulkner-Naczi K, Gonzalez-Alegre P: Attitudes of Potential Subjects Towards Molecular Therapy Trials in Huntington's Disease Journal of Huntington's Disease 8 (1): 79,2019.
Hitti FL, Gonzalez-Alegre P, Lucas TH.: Gene Therapy for Neurological Disease: A Neurosurgical Review. World Neurosurgery 121 : 2019.
Maguire JA, Gagne AL, Gonzalez-Alegre P, Davidson BL, Shakkottai V, Gadue P, French DL.: Generation of Spinocerebellar Ataxia Type 2 induced pluripotent stem cell lines, CHOPi002-A and CHOPi003-A, from patients with abnormal CAG repeats in the coding region of the ATXN2 gene. Stem Cell Research 34 : 2019.
Pedro Gonzalez-Alegre; Genevieve Beauvais; Janine Martin; Rick J. Koch; Ruth H. Walker; Jyoti C. Patel; Margaret E. Rice; Michelle E. Ehrlich.: A novel transgenic mouse model to investigate the cell-autonomous effects of torsinA(E) expression in striatal output neurons Neuroscience : 2019.
Tanya M. Bardakjian, Jennifer Klapper, Adrianna Carey, Julia Wood, Meredith Pauly, Kelly Gasper; Kathy Lawler, Baochan Tran; Yuliis Bell; Alex Zwil, Pedro Gonzalez-Alegre: Addressing the value of multidisciplinary clinical care in
Huntington's Disease: a snapshot of a new Huntington's Disease
Center Journal of Huntington Disease : 2019.
Joseph Park; Scott M. Damrauer; Aris Baras; Jeffrey G. Reid; John D. Overton; Pedro Gonzalez-Alegre.: Epidemiology of DYT1 dystonia: estimating prevalence via genetic ascertainment Neurology Genetics : 2019.
Pedro Gonzalez-Alegre.: Recent advances in molecular therapies for neurological
disease: triplet repeat disorders. Hum Mol Genet : 2019.