Additional Research
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BioFocus
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After developing an assay using the neuron-like cell line to measure the secretion of proteins upon viral 'suppression' of torsin A in Phase 1, researchers are moving on to the next step of screening the BioFocus "SilenceSelect", a library which targets the human drugable genome. In efforts to redeuce the number of hits produced by either experimental procedures or secondary effects, all targets will undergo multiple screenings as well toxicity experiments to adjust screening conditions.
In 2009, The Dystonia Medical Research Foundation and Tyler's Hope for a Dystonia Cure joined forces to launch groundbreaking medical research in hopes of discovering new drug targets. Several hundred hits for potential proteins and genes that could rescue cells from the effects fo the DYT1 dystona mutation were identified from a library of 4,500 candidates. Referred to as Phase 1, these potential proteins and genes were implemented in drug discovery programs to develop therapeutics to aleviate dystonia.
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In the last phase of the project, a narrowed down selection of validated targets will be screened again by using a different assay and cell biological methods. The goal is to identify a manageable number of validated target that are likely players in the disease process. When successfully completed this will allow for identification of genes and proteins affecting torsinA function that might become targets for rational drug design. The identified targets will also enrich our knowledge about the role of torsinA in neurons and guide future research in this area.
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Tyler's Hope " Season of Hope" Fellowship Program
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At the Center’s 5th annual Think Tank (“Novel Approaches to a Cure for DYT-1 Dystonia"), Tyler's Hope graciously donated $100,000 to the establishment of the Tyler's Hope Season of Hope Fellowship Program. Providing the funds to educate and train clinical researchers, Tyler's Hope continues to support annually one of the largest fellowship programs in the world. Supporting the restoration of hope in finding a cure, the CMD Fellowship Program supports the critical need for dystonia and Parkinson’s disease specialists in the medical field.
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UAB
CRISPR Cas9 Alignment Grant II |
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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VIB
Visiting Fellowship program |
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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