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Funded Studies

Validation of TorsinA as a Target for PD Therapy in Mammalian Models

Mutations in the protein torsinA cause a hereditary form of dystonia. While the normal function of torsinA is not entirely clear, it is thought to be a “chaperone,” a molecule which helps cell repair damaged or misfolded proteins. In a worm model of PD, increasing torsinA function is protective. Several drugs which can activate torsinA in worms have been discovered. The goal of this project is to determine whether the protective effect of torsinA is present in mice. If it is, this would enable further development of neuroprotective drugs based on the strategies used in worms.
Project Description:
This study will rely on pre-clinical models in which the expression of torsinA can be altered. Most of the kinds of mice needed have already been developed, for use in studies of torsinA itself. Using these mice, we will determine whether increasing torsinA can protect mice against models of PD, and whether reducing torsinA increases the vulnerability. Both neurotoxin (MPTP) and alpha-synuclein based models of PD will be used. 
Relevance to Diagnosis/Treatment of Parkinson’s Disease: 
If we find that increasing torsinA expression is protective in mice, as it is in the worm model, this would enable further development of drugs which enhance torsinA function as a potential neuroprotective treatment.
Anticipated Outcome: 
This study will address the question of whether or not enhancing torsinA is protective in pre-clinical models of PD. This study, which will be performed at the University of Alabama at Birmingham, was designed in collaboration with QRx Pharma, Inc. If the study suggests torsinA is effective, QRx Pharma is positioned to advance this discovery further towards clinical application.


  • David G. Standaert, MD, PhD

    Birmingham, AL United States

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