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Funded Studies

Assay-PoC for the Reduction of Endogenous Alpha-synuclein Levels in iPS-PD Neurons

Study Rationale:                             
Accumulation of the alpha-synuclein protein in the brain of individuals with Parkinson’s disease (PD) is a hallmark of the disease, and mutations in the alpha-synuclein gene are associated with PD. Reducing levels of alpha-synuclein in the brain and, specifically, in nerve cells, is a promising therapeutic strategy.

We aim to identify a novel compound that will reduce alpha-synuclein protein levels in human nerve cells.

Study Design:
We will develop a highly sensitive assay (laboratory test) to accurately quantify levels of the alpha-synuclein protein in human nerve cells. In addition, we will develop methods to culture nerve cells from individuals with Parkinson’s. The combination of these two approaches will allow us to establish a proof-of-concept study that shows how we can identify novel compounds that reduce alpha-synuclein levels in human nerve cells.

Impact on Diagnosis/Treatment of Parkinson’s Disease:                              
We believe that identifying novel compounds that safely reduce levels of the alpha-synuclein protein in the human brain could lead to the development of potentially effective treatments for PD.

Next Steps for Development:
When the proof-of-concept study is successfully completed, we will screen for novel compounds that reduce levels of the alpha-synuclein protein in human nerve cells. Additional steps include subsequent safety testing, as well as additional studies to help us better understand the mechanism of action underlying the activity of these compounds.


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