1. The Michael J. Fox Foundation's Strategies for Accelerating Translation of LRRK2 into Therapies for Parkinson Disease.
- Author
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Padmanabhan S, Fiske BK, and Baptista MAS
- Subjects
- Animals, Humans, Leucine-Rich Repeat Serine-Threonine Protein Kinase-2 genetics, Parkinson Disease genetics, rab GTP-Binding Proteins genetics, rab GTP-Binding Proteins metabolism, Biomarkers metabolism, Leucine-Rich Repeat Serine-Threonine Protein Kinase-2 metabolism, Parkinson Disease metabolism
- Abstract
Since 2005, The Michael J. Fox Foundation for Parkinson's Research (MJFF) has invested significant funding and non-funding effort to accelerate research and drug development activity around the Parkinson disease (PD)-associated protein LRRK2. MJFF has spearheaded multiple public/private pre-competitive collaborations that have contributed to our understanding of LRRK2 function; de-risked potential safety questions around the therapeutic use of LRRK2 kinase inhibitors; and generated critical research tools, biosamples, and data for the field. Several LRRK2-targeted therapies are now in human testing due to the hard work of so many in the PD community. In this perspective, we present a holistic description and model of how our Foundation's support targeted important barriers to LRRK2 research and helped move the field into clinical trials.
- Published
- 2020
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