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Preclinical and clinical resources for Parkinson's disease research

The Michael J. Fox Foundation for Parkinson’s Research (MJFF) is accelerating Parkinson’s disease (PD) research by decreasing barriers and increasing access to critical resources.   MJFF ensures that key preclinical reagents, animal models, clinical tissues, biosamples and data resulting from characterization studies, are made widely available to researchers.

MJFF is supporting the generation and widespread dissemination of a number of preclinical tools including DNA plasmids, viral vectors, protein, antibodies, assays, cell lines and animal models.  The Foundation has also supported characterization efforts around each tool that provides the community with confidence to utilize these tools in research efforts.  Publication of these results and ensuring that raw data is accessible via public databases is also essential to adequate resource development. 

To date MJFF has generated and characterized more than 25 antibodies, 20 animal models, 10 viral vectors and an assay around PD targets including alpha-synuclein, LRRK2, DJ-1, Parkin, PINK1, VPS35, Eif4g1 and glucocerebrosidase.  Furthermore, a number of other novel tools including assays, and cell lines and protein reagents are currently under development.  Since 2010, over 3,000 antibodies, 250 assays, 3,000 animal models and 1,000 viral vectors have been distributed. 

Clinically relevant resources that enable investigators to identify biological markers that may assist clinical and therapeutic efforts are also another key unmet need in the field.  Among these, diagnostic biomarkers and progression markers that track with different disease stages is critical given the inherent heterogeneity associated with PD.  The identification and qu   alification of diagnostic and prognostic biomarkers of PD would dramatically accelerate clinical research and development of disease modifying PD therapeutics that enables scientists to gauge cellular and/or molecular changes.  MJFF is collecting biologic tissues and samples (including blood, CSF, urine, DNA, RNA) from several different cohorts, including individuals with PD and control subjects. 

MJFF is confident that the availability of these reagents to the entire research community will facilitate understanding of PD and accelerate therapeutic development. Further, the Foundation can replicate this strategy for other limited resources.  In this way, MJFF strives to decrease barriers to entry for PD research efforts and promote access to critical tools.   

Authors: Sonal S. Das, Meredith Haupt, Audrey Dufour, Marco Baptista, Kuldip Dave, Catherine Kopil, Jamie Eberling, Maurizio Facheris, Adria Martig, Dario Alessi, Paul Davies, Andrew West, Peggy Taylor, Lona Vincent, Nicole Willis, Annesha White, Brennan Long, Alison Urkowitz, Brian Fiske, Todd Sherer, Mark Frasier

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