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Review Article
Rationale for Therapeutic Silencing of Alpha-Synuclein in Parkinson’s Disease
Demetrius M. Maraganore
J Mov Disord. 2011;4(1):1-7.
DOI: https://doi.org/10.14802/jmd.11001
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AbstractAbstract PDF

The purpose of this paper is to provide the rationale for therapeutic silencing of the alpha-synuclein gene (SNCA) in Parkinson’s disease (PD). The paper reviews the public health significance of PD; the causal links between rare SNCA variants and familial PD; the association of common SNCA variants and PD susceptibility; the association of SNCA variants also with age at onset and motor and cognitive outcomes in PD; therapeutic strategies targeting SNCA in PD; and preliminary findings and considerations on small interfering RNA-based therapies and PD.

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