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Funded Studies

Development of a Novel Intracellular Alpha-synuclein-lowering Strategy based on CRISPR Interference Technology

Study Rationale:
Alpha-synuclein protein accumulates in the brain in Parkinson’s disease. Reducing the impact of this protein in the brain may prevent the degeneration of nerve cells and stop the progression of the disease.

Our objective here is to test the hypothesis that reducing the production of alpha-synuclein in diseased brain cells will help improve their survival and function.

Study Design:
We will graft human dopamine neurons prepared in a laboratory dish into the brain of models and administer viral vectors to introduce genes that will control and reduce the expression of alpha-synuclein in the neurons. We know that the grafted human dopamine neurons gradually develop the pathology we normally see in the brain (thus replicating the human disease in model brain). The main outcomes here would be to assess how well we can reverse these pathologies and keep the cells alive as we apply and test our novel gene therapy approach.

Impact on Diagnosis/Treatment of Parkinson’s Disease:
When successful, this study will have two types of impact. First, it will demonstrate that reducing the alpha-synuclein burden on diseased neurons is beneficial for them. Secondly, it will allow us to define more precisely the strategy and the sequences of the genetic constructs we could take forward to testing in humans in the next phase.

Next Steps for Development:
The steps immediately following this work will be two-fold: (1) test the validity of the approach in a larger model and show that the results are scalable to targeting larger brains; (2) design the clinical candidate construct and plan the pre-clinical testing of that precise gene therapy product toward approval for testing in humans.


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