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Funded Studies

Targeting RER1 in Models of Parkinson's Disease

Study Rationale:
Abnormal build-up of the protein alpha-synuclein in Parkinson's disease (PD) has been linked to cell stress and death. These deficits can be rescued by expressing certain proteins that affect endoplasmic reticulum-golgi (production and sorting of proteins and lipids) trafficking (movement across the cell). Increasing evidence suggests that the Retention in endoplasmic reticulum 1 (RER1) protein plays a key role in regulating protein trafficking and development. We have recently shown that RER1 expression reduces alpha-synuclein levels in cell and neuron cultures.

We hypothesize that RER1 plays an important role in regulating alpha-synuclein breakdown when levels are high and that overexpression will prevent alpha-synuclein build-up and associated pathology in Parkinson's models.

Study Design:
We plan to test whether viral expression of RER1 in the brain (or spinal cord) can reduce alpha-synuclein pathology, including clumping and neurodegeneration, in a pre-clinical model of parkinsonism.

Impact on Diagnosis/Treatment of Parkinson's Disease:
The findings from these studies should lead to a better understanding of the role of RER1 in alpha-synuclein processing and breakdown. The results may also impact the development of new drugs targeting RER1 or other molecules that mediate protein transport and levels of alpha-synuclein in Parkinson's disease.

Next Steps for Development:
If successful, the results of these studies will stimulate further tests targeting RER1 or other molecules for novel therapies aimed at reducing alpha-synuclein pathology in Parkinson's.


  • Nikolaus McFarland, MD, PhD

    Gainesville, FL United States

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