title: Rationale for therapeutic silencing of alpha-synuclein in Parkinson's disease.
abstract: 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.
authors.affiliation: Ruth Cain Ruggles Chairman, Department of Neurology, and Medical Director, Neurological Institute, NorthShore University Health System, Evanston, IL, USA. Clinical Professor of Neurology, Pritzker School of Medicine, University of Chicago, Chicago, IL, USA.
authors.forename: Demetrius M
authors.initials: DM
authors.lastname: Maraganore
keywords: Alpha-synuclein; Parkinson’s disease; RNA-based therapies