Genome Wide Analysis of Alpha-Synuclein Neurotoxicity

NIH RePORTER · NIH · R01 · $585,729 · view on reporter.nih.gov ↗

Abstract

Parkinson's disease is the most common neurodegenerative movement disorder and is characterized pathologically by the intraneuronal deposition of abnormally phosphorylated and aggregated α-synuclein protein. Abnormal deposition of α-synuclein into neuronal and glial aggregates is also the primary pathologic feature of a group of collectively even more common disorders, termed the α-synucleinopathies. To define the molecular mechanisms controlling α-synuclein induced neurodegeneration we and others have modeled α-synucleinopathies in the simple and powerful genetic model organism Drosophila. Genetic, biochemical and cell biological experiments in Drosophila have provided important clues regarding the pathogenesis of α-synucleinopathies. However, the unbiased forward genetic screens providing the bases for these studies, while valuable, have to date remained incomplete. Here we propose to use a newly created and powerful Drosophila model of α-synucleinopathies to perform a comprehensive genetic analysis of α-synuclein neurotoxicity in vivo. These studies will for the first time provide a broad analysis of mechanisms controlling α-synuclein toxicity to postmitotic neurons and should identify many new high-value therapeutic targets. Our studies will be particularly important as more and more data emerges from genome wide associated studies showing genetic influences on Parkinson's disease and related α-synucleinopathies, but with little clear evidence as to the mechanism of action of these newly identified gene products in neurodegenerative disease pathogenesis.

Key facts

NIH application ID
9995036
Project number
5R01NS098821-04
Recipient
BRIGHAM AND WOMEN'S HOSPITAL
Principal Investigator
MEL B FEANY
Activity code
R01
Funding institute
NIH
Fiscal year
2020
Award amount
$585,729
Award type
5
Project period
2017-09-25 → 2022-06-30