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.

SUBMITTER: Maraganore DM 

PROVIDER: S-EPMC4027709 | biostudies-literature | 2011 May

REPOSITORIES: biostudies-literature