Research at UF
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Tyler’s Hope Center of Excellence
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The UF Center for Movement Disorders is designated the first Tyler’s Hope Center of Excellence. The Movement Disorders Center has been a leader in dystonia research through their innovative clinical care, novel research approaches, dystonia fellowship program, and doctors dedicated to finding a cure while providing the most advanced treatments available. Because of the partnership and focus on dystonia care the Center for movement disorders (Center of Excellence) is recognized as the world’s leading dystonia center.
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Bachman Strauss Center of Excellence
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On September 26, 2013, Tyler’s Hope Foundation funded the opening of the Bachmann-Strauss Center for Excellence at the UF Center for Movement Disorders. Supporting scientific and clinical research for dystonia and Parkinson’s disease, the Bachmann-Strauss Center for Excellence continues to spread awareness to the general public and medical community in hopes of finding a cure.
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In less than a year, the opening of the Bachman-Strauss Center for Excellence has supported fundamental breakthroughs and discoveries in the pathophysiology of DYT1 dystonia and potential gene and protein targets for torsion A dysfunction.
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Torsin A Study - Dr. Robert Mckenna, Dr.Li, Dr. Shyamasri Biswas
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To better understand the enhancemnet of torisinA function in vivo caused by ampicillin, researchers have investigated the structure and biophysical characterization of torsinA. Further investigation of the structure will also determine the ph and thermal stabilitiy properties of torsinA. Moving closer to the underlining cause of dystonia, this study has provided numerous oppurtunities for the pathophysiology and neural interferance of dystonia.
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Results from this study have shown potential breakthroughs and discoveries for the development of drugs for dystonia therapy. Pin-pointing several small molecules repsonsible for enhancing normal wild-type torsin A activity, researchers shed light for potential reduction of mutant torsin A activity in early onset dystonia.
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Dr. Li
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In 2010, UF Center for Movemet Disorders and Neurorestoration had the privelege of recruiting the country's most promising dystonia researcher, Dr. Yuqing Li to the University of Florida. After raising one million dollars, the center constructed a state-of-the-art laboratory for dystonia research.
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Ampicillan Trial - Dr. Rodriguez
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Early onset torsional dystonia is normally caused by the deletion of glutamic acid in the DYT1 gene product, torsionA. Causing mutants in torsionA, such deffects result in a decrease torsion A function. In hopes of restoring normal cellular functionality, representative molecules such as aminopenicillin and quinolones were analyzed in EOTD patient fibroblasts for improvements in torsion A dependent secretory function. Increasing the activity of torsion A activity, ampicillin was linked to reduced behavioral defectsin knock-in mouse models. Treamtent of ampicilllin in the DYT1 gene mutation showed signs of improved motor coordination.
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At the University of Florida's MDC, researchers are conducting a study to look at safety and tolerability of ampicillin to improve dystonia symptoms under the clincial trial.
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Fellowship Program
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Tyler’s Hope is dedicated to supporting the UF Center for Movement Disorder’s fellowship program. Through this collaboration the clinic’s fellowship program has been recognized as the leading movement disorders program, producing over 20% of the nation’s movement disorder fellows. Each fellow is required to learn and work with dystonia patients.
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Visiting Fellowship Program
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The VIB at KU – Leuven in Belgium has an excellent dystonia research center lead by group leader Dr. Rose Goodchild. The research is focused on the biology of dystonia. The VIB does not have a clinical program in which the fellows working on dystonia have a chance to work with patients and see first-hand the effects of DYT1 dystonia. Teaming up with the UF Movement Disorders Center, the VIB will send fellows annually to visit the UF Center and get a chance to participate in clinical rotations. The fellows will also have the opportunity to participate in the Tyler’s Hope Think Tank at the end of the program.
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CRISPR Cas9 Alignment Grant II
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The CRISPR Cas 9 alignment grant project is a collaboration project between the institutions: UF, UAB, and Harvard. This project will specifically target the DYT1 gene producing the mutant protein that causes the symptoms of dystonia. The theory is that by introducing a therapy that deletes the genetic code producing the mutation, those with dystonia will stop manufacturing the "bad form" of the Torsin protein and your body will function normally with the "good form" of the protein already being produced.
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