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Funded Studies

Stem Cells with Different Amounts of Alpha-synuclein as a Drug Discovery Tool

Promising Outcomes of Original Grant:
Stem cells are a renewable source of tissue that can be coaxed to become different cell types of the body. When prompted, stem cell models can become neural cells. Previously, we successfully developed several stem cell models by altering skin cells from people with Parkinson's disease (PD). We then gave these stem cells the ability to make precise amounts of alpha-synuclein -- the sticky protein that clumps in the brains of people with PD -- by controlling activity of alpha-synuclein genes. The level of markers, or indicators, of Parkinson's disease in these cell models depends on the amount of alpha-synuclein each model produces. Previous studies have shown that slowing the production of alpha-synuclein or removing harmful clumps from neural cells may help slow or stop Parkinson's disease progression.

Objectives for Supplemental Investigation:
We now aim to develop additional stem cell models. Once additional models are developed, we will evaluate all the models to identify their similarities and differences and any possible negative side effects of DNA alteration. The varying amount of alpha-synuclein -- from none to two-fold excess -- in each model produces a different effect. We will study the effect of alpha-synuclein on these models when they become neural cells by measuring Parkinson's markers in these cells. Lastly, we will evaluate the overall health of these cells and their ability to form networks, a particularly important property of the neural cells. All stem cell models will be deposited in a cell bank for storage and made available to the research community at the end of the study.

Importance of This Research for the Development of a New PD Therapy:
Slowing the production of alpha-synuclein or removing harmful clumps from neural cells may help prevent, slow or stop Parkinson's disease progression. Alpha-synuclein has become a leading therapeutic target for this reason. Our unique stem cells can model the effects of lower alpha-synuclein levels in the cell, and these models could become valuable drug discovery tools in PD research.


Researchers

  • Birgitt Schüle, MD, Dr. med.

    Stanford, CA United States


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