Rationale for Therapeutic Silencing of Alpha-Synuclein in Parkinson’s Disease

• Main Author: Demetrius M. Maraganore
• Format: Article
• Language: English
• Published: Korean Movement Disorders Society 2011-05-01
• Series: Journal of Movement Disorders
• Subjects: Parkinson’s disease; Alpha-synuclein; RNA-based therapies
• Online Access: http://e-jmd.org/upload/jmd-4-1-1-1.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.