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Validation of VPS41, a Protein Involved in Lysosomal Trafficking, as a Target for Parkinson Disease Therapy

Promising Outcomes of Original Grant:
VPS41 was identified in a C. elegans system. In the original funded grant, we demonstrated that human VPS41 (hVPS41) was also protective in C. elegans, performed a domain analysis and identified a minimally protective structure, constructed mammalian cells lines stably transfected with hVPS41 and validated protection against PD-relevant neurotoxins, observed inhibition of accumulation of alpha-syn aggregates, generated a knockout mouse, and produced an AAV viral vector for VPS41 expression.

Objectives for Supplemental Investigation
Progress on the pre-clinical model studies proposed in the original grant were delayed by difficulties genotyping the knockout, the need to breed congenic animals, and delays in production of the AAV vector. This supplement will allow us to complete the originally proposed aims. 

Importance of This Research for the Development of a New PD Therapy:
VPS41, discovered in C. elegans, exhibits strong protective properties in mammalian cells. It is one of a few molecules known to be effective in both neurotoxin and alpha-syn assays. Validation in pre-clinical models is critical for advancement as a target for PD therapy.


  • David G. Standaert, MD, PhD

    Birmingham, AL United States

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