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Funded Studies

RNA Targeting Small Molecules as Therapeutics for Parkinson’s

Study Rationale:    
Abnormal clumping of the protein alpha-synuclein is a hallmark of Parkinson's disease (PD) and a likely cause of neurodegeneration and, ultimately, symptoms. One of the causes that can speed up this clumping is the amount of this protein in the brain. Therefore, finding ways to reduce how much of this protein is produced in the brain may slow disease progression. The goal of this study is to identify compounds that can travel to the brain and reduce the amount of alpha-synuclein and test their efficacy in the laboratory.

Hypothesis:
We hypothesize that small molecule compounds that we can design have the ability to reduce the production of alpha-synuclein, minimize its clumping, and prevent or slow down brain cell damage.

Study Design:
New compounds that have the ability to reduce alpha-synuclein production will be evaluated, and the best candidates will be tested in pre-clinical models with Parkinson's features. The impact of this treatment on the ability of alpha-synuclein to clump and damage the brain will be assessed.

Impact on Diagnosis/Treatment of Parkinson’s disease:        
Identifying small molecule compounds that prevent or minimize the abnormal clumping of alpha-synuclein can help develop a disease-modifying treatment for PD.

Next Steps for Development:
If the efficacy of a small molecule compound is demonstrated in the pre-clinical models of PD, we will develop this treatment further and test it in clinical trials.
 


Researchers

  • Mary Maral Mouradian, MD

    Piscataway, NJ United States


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