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The long-range goal of research in the Gonzalez-Alegre Lab is to develop treatments for human diseases caused by basal ganglia dysfunction based on biological information gained from studies performed in cellular and animal systems and in patients seen in the clinic. The lab focuses on the study of inherited diseases, mostly dystonia, Huntington’s Disease and inherited ataxias.
The lab’s research on inherited dystonia uses cellular and animal models to better understand the neurobiological process underlying this movement disorder. By investigating different genetic forms of this disorder, such as DYT1 and DYT6, the research team aims to identify shared pathogenic pathways, with the goal of devising therapeutic strategies that could be applicable to different forms of dystonia. In addition, the Gonzalez-Alegre Lab is developing gene silencing strategies, such as RNAi and antisense oligonucleotides, as potential therapies for specific genetic forms of dystonia.
Through collaborative translational work with other member of the Center for Cell and Molecular Therapy, Dr. Gonzalez-Alegre directs the efforts to design and perform early phase clinical trials in patients with neurological disease using molecular therapeutic approaches such as gene replacement or silencing.
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.