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New Frontiers in Parkinson's Disease: From Genetics to the Clinic.


ABSTRACT: The greatest unmet therapeutic need in Parkinson's disease (PD) is a treatment that slows the relentless progression of the symptoms and the neurodegenerative process. This review highlights the utility of genetics to understand the pathogenic mechanisms and develop novel therapeutic approaches for PD. The focus is on strategies provided by genetic studies: notably via the reduction and clearance of ?-synuclein, inhibition of LRRK2 kinase activity, and modulation of glucocerebrosidase-related substrates. In addition, the critical role of precompetitive public-private partnerships in supporting trial design optimization, overall drug development, and regulatory approvals is illustrated. With these great advances, the promise of developing transformative therapies that halt or slow disease progression is a tangible goal.

SUBMITTER: Shihabuddin LS 

PROVIDER: S-EPMC6705997 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature

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New Frontiers in Parkinson's Disease: From Genetics to the Clinic.

Shihabuddin Lamya S LS   Brundin Patrik P   Greenamyre J Timothy JT   Stephenson Diane D   Sardi S Pablo SP  

The Journal of neuroscience : the official journal of the Society for Neuroscience 20181001 44


The greatest unmet therapeutic need in Parkinson's disease (PD) is a treatment that slows the relentless progression of the symptoms and the neurodegenerative process. This review highlights the utility of genetics to understand the pathogenic mechanisms and develop novel therapeutic approaches for PD. The focus is on strategies provided by genetic studies: notably via the reduction and clearance of α-synuclein, inhibition of LRRK2 kinase activity, and modulation of glucocerebrosidase-related su  ...[more]

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