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

oleh: Demetrius M. Maraganore

Format: Article
Diterbitkan: Korean Movement Disorder Society 2011-05-01

Deskripsi

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.