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Funded Studies

Targeting the Stress Sensor PERK for the Treatment of Parkinson´s Disease

Objective/Rationale:             
Parkinson's disease (PD) is characterized by impairment of motor control as a result from extensive neuron death. The primary mechanism responsible for the progressive neuronal loss in PD remains unknown. It has been suggested that perturbation in the function of a subcellular organelle called the endoplasmic reticulum (ER) may determine the pathological effects of PD. In this project we aim to define the direct contribution of this stress pathway to PD using models of the disease. We intend to define the possible therapeutic benefits of alleviating cellular stress using two novel drugs.

Project Description:
By employing two novel compounds that were previously validated in other neurodegenerative diseases, we will manipulate the activity of an essential ER stress pathway known as PERK. These novel drugs show great specificity and favorable pharmacokinetic and safety properties.

Relevance to Diagnosis/Treatment of Parkinson’s Disease:                     
This work will help validate a novel therapeutic target and may foster development of a new therapy to stop the progression of Parkinson’s disease.

Anticipated Outcome:          
We expect to reduce the loss of dopaminergic neurons in two models of PD and to improve motor performance after administrating the ER stress-targeting drugs.


Researchers

  • Claudio Hetz Flores, PhD

    Santiago Chile


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