Work

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

Year: 2011

Type: review

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.

Source: Journal of Movement Disorders

DOI: 10.14802/jmd.11001

Author: Demetrius M. Maraganore

Institutions: Endeavor Health, University of Chicago

Language: English

Topic: Parkinson's Disease Mechanisms and Treatments

Subfield: Clinical Neurology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: diamond

Funders: National Institutes of Health, National Institute of Neurological Disorders and Stroke, National Institute of Environmental Health Sciences