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Screening Assays for Restored Protein Trafficking

Study Rationale:
Protein trafficking is the process by which proteins are transported from where they’re made to where they’re needed inside cells. Defects in trafficking cause proteins to accumulate inside cells, and this puts the cells under stress. In the case of neurons, this stress can lead to neuronal death. We are applying a technology that allows us to track proteins inside cells to create a screen for compounds that restore proper trafficking, relieving neurons from this extra stress and hopefully preventing neurodegeneration.

We believe that our Fluorogen Activating Peptide technology can be used to create a high-speed drug screening assay (experiment) to search for compounds that will restore proper trafficking of mutated Glucocerebrosidase (GCase), a protein trafficking defect highly correlated with Parkinson’s disease.

Study Design:
Using our trafficking assay technology, we will create cells that model the trafficking defect seen in humans. We will create cells that have normal GCase, and cells that contain the most common GCase mutations. We will then verify defective trafficking of the mutants, and normal trafficking for normal GCase. This assay will become the basis for a high-speed drug screening assay. 

Impact on Diagnosis/Treatment of Parkinson’s Disease:             
GCase mutation is one of the most common risk indicators for Parkinson’s. A drug that corrects this defect could be used in patients that test positive for these mutations, yielding a truly targeted, personalized Parkinson’s therapy.

Next Steps for Development:
Following successful development of the drug screening assay, we will progress to screening compound libraries for potential lead compounds as part of a drug discovery effort. We plan to also make these assays available for use by other researchers.


  • Scott F. Sneddon, PhD, JD

    Pittsburgh, PA United States

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